U.S. Food and Drug Administration

Receiving rolling BLA submission for lonvo-z under RMAT designation; full submission expected H2 2026

On April 27, 2026, Intellia Therapeutics reported that its Phase 3 HAELO trial of lonvoguran ziclumeran (lonvo-z) — a one-time, in-body CRISPR gene-editing therapy for hereditary angioedema (HAE) — met its primary endpoint and all key secondary endpoints. In the 80-patient, randomized, double-blind trial, a single intravenous infusion of lonvo-z cut the rate of debilitating swelling attacks by 87% compared with placebo in the primary observation window (weeks 5 through 28). Sixty-two percent of patients who received the therapy were completely attack-free and had stopped all preventive medications in that period. The safety profile was clean: all reported side effects were mild or moderate, with no serious adverse events, and infusion-related reactions, headache, and fatigue were the most common complaints.

Updated 14 hours ago

Future regulator for zasocitinib’s NDA; the main gatekeeper for U.S. commercialization

Psoriasis has been an injectable kingdom for years: shots that work great, pills that usually don't. Takeda's once-daily TYK2 pill zasocitinib just cleared Phase 3 topline hurdles—and by the next session investors responded, sending Takeda shares up as much as 4.3% in early Tokyo trading (a seven-month-high intraday jump).

Updated Yesterday

Defendant; defending 2021 rule changes

The Supreme Court has until Thursday at 5 p.m. ET to decide whether mifepristone can keep shipping by mail. Alito's stay, expiring May 14, is all that blocks a May 1 Fifth Circuit ruling that would end telehealth prescribing and mail delivery of the drug nationwide.

Updated 3 days ago

Reviewing mRNA-1010 with August 5, 2026 decision deadline

For 80 years, flu vaccines grew in chicken eggs — a process effective against roughly 40% of infections in good years. On May 8, the New England Journal of Medicine published Moderna's Phase 3 data: mRNA-1010 reduced influenza illness by 26.6% more than a standard shot in 40,805 adults aged 50 and older. Moderna's stock rose 16% that day.

Updated 5 days ago

Primary regulator of AI medical devices in the United States

In February 2026, University of Michigan researchers published Prima, an AI that reads brain MRIs in seconds and flags emergencies with up to 97.5% accuracy across 50+ neurological conditions. The study arrived as the U.S. faces a radiologist shortage projected to last through 2055 and the FDA has cleared over 1,100 AI-enabled radiology devices. Three months later, Prima still awaits FDA review.

Updated 5 days ago

Has granted Breakthrough Device Designation to multiple decongestion devices

A catheter threaded into a vein in the neck pulled an average 6.8 kilograms of excess fluid out of 40 hospitalized heart failure patients without damaging their kidneys. Six months later, 82.5 percent were still alive and out of the hospital.

Updated 6 days ago

Granted approval

Patients with advanced bile duct cancer driven by a rare genetic fusion called NRG1 had nothing approved for them after chemotherapy failed. On May 8, the U.S. Food and Drug Administration (FDA) approved Bizengri (zenocutuzumab-zbco), made by Partner Therapeutics, as the first drug aimed at this small group. It works by blocking the abnormal protein the NRG1 fusion produces, which fuels tumor growth.

Updated 7 days ago

Approved Lerochol (December 2025); evaluating enlicitide decanoate as potential first oral PCSK9 inhibitor

For two decades, lowering LDL cholesterol meant statins—daily pills that work for most but leave roughly one in five patients with muscle pain or liver enzyme changes severe enough to quit. On May 1, researchers at the University of Barcelona and Oregon Health & Science University published results on a short, engineered DNA molecule that silences the gene controlling how the liver clears LDL, cutting cholesterol by nearly 50 percent in mice after a single injection.

Updated May 7

Accepted Helix pre-submission for substantive review; Q2 2026 meeting scheduled; reviewing CardiAMP accelerated approval request

BioCardia's campaign to win the first FDA clearance for a catheter that injects cell and gene therapies directly into heart muscle cleared a pivotal regulatory hurdle in March 2026. The U.S. Food and Drug Administration accepted the company's pre-submission package for the Helix Transendocardial Delivery Catheter, confirming it contains all the necessary elements for substantive review and scheduling a formal meeting for the second quarter of 2026. The Helix device, backed by safety and efficacy data from fifteen clinical trials, uses a small helical needle that anchors within the beating heart to precisely deliver therapeutic agents to damaged muscle tissue. CDRH will lead the review in consultation with CBER.

Updated May 7

Reviewing safety and efficacy of next-generation candidates

Boys born with Duchenne muscular dystrophy typically lose the ability to walk before their teens and rarely live past 30. The first drugs designed to slow that decline reached the market in 2016, but they restore less than 1% of normal dystrophin, the muscle protein patients are missing. A decade later, a new wave of biotech companies is trying to deliver far more of that protein into muscle cells using engineered molecular shuttles.

Updated May 7

Approved supplemental application expanding ASCENIV's pediatric indication

For seven years, an immune therapy made from antibody-rich human plasma was available only to teenagers and adults. On May 4, 2026, the U.S. Food and Drug Administration extended ADMA Biologics' ASCENIV to children as young as two—closing a gap that left some of the most vulnerable pediatric patients dependent on off-label prescribing or alternative drugs.

Updated May 4

Pre-NDA meeting with Mirum scheduled for summer 2026

For the roughly 30,000 Americans living with primary sclerosing cholangitis (PSC), the U.S. Food and Drug Administration has never approved a single drug. On May 4, 2026, that drought moved closer to ending: Mirum Pharmaceuticals reported that its experimental pill volixibat hit the primary endpoint of its Phase 2b VISTAS trial, cutting the relentless itch that defines the disease by a wide margin over placebo.

Updated May 4

Approved first-in-class PROTAC 35 days early

For 25 years, drug companies have tried to design medicines that don't just block harmful proteins but destroy them outright. On May 1, the U.S. Food and Drug Administration (FDA) approved the first one. VEPPANU, developed by biotech Arvinas and Pfizer, treats a hard-to-treat form of metastatic breast cancer by hijacking the cell's own garbage-disposal system to shred a mutated estrogen receptor that fuels the tumor.

Updated May 1

Issued approval on PDUFA action date

Until May 2023, no drug was approved in the United States to treat the agitation that affects up to three-quarters of people with Alzheimer's disease. Doctors reached for antipsychotics off-label, a class of medicines that carries a black-box warning for increased death risk in older dementia patients. On April 30, 2026, the Food and Drug Administration (FDA) approved Axsome Therapeutics' Auvelity for the indication, doubling the menu of approved options from one to two. In its own press announcement, the FDA described the action as approving the 'first non-antipsychotic drug to treat agitation associated with dementia' — a framing that underscores what distinguishes Auvelity from the incumbent treatment, Rexulti.

Updated May 1

Co-issuing RAPID pathway

For nearly a decade, medical device makers have faced the same bottleneck: the FDA clears a breakthrough device, and then Medicare spends another year or more deciding whether to pay for it. On April 23, 2026, the two agencies jointly announced the Regulatory Alignment for Predictable and Immediate Device (RAPID) pathway to collapse that gap to as little as two months — and simultaneously paused the existing Transitional Coverage for Emerging Technologies (TCET) program for new applicants, consolidating all breakthrough-device coverage work under the new pathway.

Updated Apr 24

Approved Yartemlea after extended review process

For decades, patients who developed transplant-associated thrombotic microangiopathy (TA-TMA) after stem cell transplants faced a grim reality: a life-threatening complication with no approved treatment. Mortality rates for high-risk cases reached 80-90%. In December 2025, the FDA approved Yartemlea, the first therapy for this condition, marking Seattle-based Omeros Corporation's first drug approval after 31 years. The company launched commercially on January 2, 2026, announced pricing at $36,000 per vial on January 7, and by late January both adult and pediatric patients were receiving treatment at transplant centers nationwide.

Updated Apr 23

Responding to executive order: issuing priority review vouchers for psilocybin-class drugs and clearing path for first US ibogaine trials

For 55 years, the federal government classified psilocybin, MDMA, LSD, and ibogaine as Schedule I substances — drugs with no accepted medical use. On April 18, 2026, President Trump signed an executive order titled 'Accelerating Medical Treatments for Serious Mental Illness,' directing the Food and Drug Administration (FDA) to expedite clinical trials of those same substances for treating post-traumatic stress disorder (PTSD) in veterans. The order, whose signing ceremony included podcaster Joe Rogan and former Navy SEAL Marcus Luttrell, was driven substantially by Health and Human Services Secretary Robert F. Kennedy Jr. It makes $50 million in federal funding through the Advanced Research Projects for Health (ARPA-H) available for state-level ibogaine research — matched by state funds — and extends the Right to Try law to allow seriously ill patients to access psychedelics still under investigation. Within hours of the signing, FDA Commissioner Marty Makary announced that the agency would issue 'national priority' review vouchers for three psilocybin-class drugs, a first for any psychedelic substance, with decisions possible as early as summer 2026. The FDA also announced steps to clear the way for the first-ever US human trials of ibogaine.

Updated Apr 19

Granted traditional approval to tarlatamab in November 2025

For decades, patients with extensive-stage small-cell lung cancer who relapsed after chemotherapy had almost nowhere to turn — five-year survival rates sat around 3%. Now a drug that physically bridges the patient's own immune cells to their tumor cells has been approved in both the United States and China, opening a new treatment class for one of the deadliest cancers. Tarlatamab, sold as Imdelltra, reduced the risk of death by 40% compared to standard chemotherapy in a confirmatory trial of 509 patients.

Updated Apr 13

Cleared the first human trial of epigenetic reprogramming therapy

In 2006, Shinya Yamanaka showed that four genes could rewind an adult cell all the way back to an embryonic-like state. Twenty years later, a stripped-down version of that technique is being injected into human eyes for the first time. Life Biosciences, a Boston biotech co-founded by Harvard aging researcher David Sinclair, has begun dosing glaucoma patients with ER-100, a gene therapy that delivers three of Yamanaka's four reprogramming factors to retinal cells — with a built-in off switch controlled by the common antibiotic doxycycline.

Updated Apr 9

Approved Foundayo under new fast-track voucher program

For two years, the most effective weight-loss drugs on the market required a weekly injection. That barrier is now falling. Eli Lilly began shipping Foundayo — the first oral GLP-1 weight-loss pill that can be taken any time of day without food or water restrictions — on April 6, 2026, just five days after the Food and Drug Administration (FDA) approved it in a record 50-day review. Priced at $149 per month for self-pay patients and as low as $25 with commercial insurance, Foundayo undercuts the list price of injectable alternatives by roughly 85%.

Updated Apr 6

Approves fourth obesity drug in 13 months: Imcivree (rare), high-dose Wegovy, oral semaglutide, and Foundayo (common)

For patients who gained severe, uncontrollable weight after brain tumor surgery—often as children—there has never been an approved medication. That changed on March 19, 2026, when the Food and Drug Administration (FDA) approved Imcivree (setmelanotide) for acquired hypothalamic obesity, a condition affecting roughly 10,000 Americans whose damaged hypothalamus makes diet and exercise essentially useless. In clinical trials, patients lost 18.4% more body mass than those on placebo over one year. Two weeks later, on April 1, the FDA approved Eli Lilly's Foundayo (orforglipron), the first GLP-1 pill taken without food restrictions, achieving 12.4% weight loss.

Updated Apr 1

Granted RMAT designation to CB-011

Every approved cancer cell therapy on the market today requires the same costly bottleneck: extracting a patient's own immune cells, engineering them in a lab over weeks, and infusing them back — a process that costs over $400,000 and leaves some patients waiting so long their cancer outpaces the manufacturing. On March 31, 2026, the Food and Drug Administration (FDA) granted accelerated regulatory status to CB-011, the first donor-derived cell therapy engineered to hide from a patient's immune system, after it produced a 92% response rate in a Phase 1 trial for multiple myeloma — a blood cancer that kills roughly 12,000 Americans each year.

Updated Mar 31

Reviewing relutrigine NDA under priority review

Children with SCN2A and SCN8A developmental and epileptic encephalopathies have never had a drug designed for their condition. They cycle through four or more medications, none approved for their specific genetic disorder, while their seizures persist and their development regresses. Now, for the first time, a targeted therapy has cleared every major hurdle on its way to the U.S. Food and Drug Administration (FDA): the agency accepted Praxis Precision Medicines' application for relutrigine and granted it priority review, setting a decision deadline of September 27, 2026.

Updated Mar 30

Approved Awiqli after initially issuing rejection in 2024

For 104 years, every insulin on the market has required at least one injection per day. On March 26, the Food and Drug Administration (FDA) approved Awiqli, a once-weekly basal insulin made by Novo Nordisk, for adults with type 2 diabetes — cutting the number of basal insulin injections from 365 to 52 per year.

Updated Mar 28

Overseeing GLP-1 post-market safety surveillance

More than 30 million Americans now take GLP-1 receptor agonist drugs like Ozempic and Wegovy to lose weight. A persistent concern has dogged the medications since their rise: that they burn through muscle along with fat, leaving patients weaker even as they get thinner. A study published in Cell Reports Medicine in March 2026 found that while GLP-1 drugs do reduce lean body mass, the rate of muscle loss is not disproportionate to what occurs with diet and exercise alone — and crucially, patients' actual strength remained unchanged.

Updated Mar 28

Granted accelerated approval to AVLAYAH

For decades, the blood-brain barrier has blocked nearly all large-molecule drugs from reaching the brain — a biological wall that left most neurological diseases effectively untreatable with modern biologics. On March 25, 2026, the Food and Drug Administration (FDA) approved AVLAYAH, developed by Denali Therapeutics, making it the first drug in history specifically engineered to cross that barrier and deliver a therapeutic enzyme to the central nervous system after a simple intravenous infusion.

Updated Mar 25

Granted expanded indication for OmniaSecure

For decades, implantable defibrillators have saved lives by shocking dangerously fast heart rhythms back to normal — but the leads that deliver those shocks also pace the heart in an unnatural pattern that can itself cause damage over time. Medtronic's OmniaSecure lead, now cleared by the Food and Drug Administration (FDA) for placement in the left bundle branch area, is the first defibrillation lead that can also pace through the heart's own electrical wiring, closely mimicking natural heartbeats.

Updated Mar 23

Will review Pfizer's Biologics License Application for the Lyme vaccine in 2026-2027

The last Lyme disease vaccine was pulled from the market in 2002 after unfounded safety fears destroyed demand. Twenty-four years later, Pfizer and French biotech Valneva announced that their replacement candidate showed 73% efficacy in a Phase 3 trial of over 9,000 participants, and said they will seek Food and Drug Administration (FDA) approval this year with a possible launch in late 2027.

Updated Mar 23

Removed psychiatric risk warnings from GLP-1 labels in January 2026

Drugs originally designed to control blood sugar and reduce weight may also protect the brain. A study of 95,490 people in Sweden, published in The Lancet Psychiatry on March 22, 2026, found that users of semaglutide—the compound in the blockbuster drugs Ozempic and Wegovy—experienced 42% fewer psychiatric hospitalizations, 44% less worsening depression, and 47% fewer substance-use-related crises compared to periods when they were not taking the drug.

Updated Mar 22

Faces pressure to revise statin labels

More than 200 million people worldwide take statins for heart health. For decades, package inserts have warned about 66 potential side effects, from memory loss to depression to muscle pain. The February 2026 Lancet analysis of 23 major clinical trials with nearly 155,000 participants found 62 of those warnings unsupported by evidence, with symptoms appearing equally in statin and placebo groups.

Updated Mar 19

Approved ICOTYDE for moderate-to-severe plaque psoriasis

For two decades, the most effective treatments for moderate-to-severe psoriasis required needles — self-injected drugs or clinic infusions that many patients refused or abandoned. On March 18, 2026, the Food and Drug Administration (FDA) approved ICOTYDE, a once-daily pill that blocks the interleukin-23 (IL-23) receptor with the precision of an injectable biologic. In clinical trials of roughly 2,500 patients, about 70 percent achieved clear or almost clear skin at 16 weeks, with side effects within 1.1 percentage points of a placebo.

Updated Mar 18

Reviewing antibiotic candidates under expedited pathways

No genuinely new class of antibiotic has reached patients since 1987. In the nearly four decades since, bacteria have steadily evolved resistance to existing drugs, and carbapenem-resistant Acinetobacter baumannii—a hospital-acquired pathogen that kills up to 60 percent of ventilated pneumonia patients—now sits atop the World Health Organization's list of critical-priority threats. On March 16, 2026, Swiss biotech BioVersys received clearance from the United States Food and Drug Administration (FDA) to begin enrolling American patients in a Phase 3 pivotal trial of BV100, a drug that cut 28-day mortality in half during earlier testing.

Updated Mar 16

Approved PureSee without contrast sensitivity warning based on clinical data

For a decade, patients choosing premium lenses during cataract surgery have faced a trade-off: better range of vision, but dimmer, lower-contrast sight—especially in low light. On March 12, 2026, the Food and Drug Administration approved Johnson & Johnson's TECNIS PureSee, the first extended depth of focus lens cleared in the United States without a warning for loss of contrast sensitivity. In clinical trials, 97% of patients reported no bothersome visual disturbances like halos or glare.

Updated Mar 12

Approved leucovorin for rare condition; declined autism indication

In September 2025, White House officials told parents of autistic children that a cheap, generic drug called leucovorin might improve their children's speech and behavior. Prescriptions surged 71% in the following months, pharmacies ran dry, and the Food and Drug Administration (FDA) allowed emergency imports from Canada and Spain. On March 10, 2026, the FDA approved leucovorin — but only for a genetic condition so rare that fewer than 50 cases have ever been identified worldwide, not for autism.

Updated Mar 10

Granted accelerated approval for Ojemda; confirmatory Phase 3 trial ongoing

For decades, children with relapsed brain tumors had no approved targeted treatment. That changed in April 2024 when the Food and Drug Administration (FDA) cleared Ojemda, a once-weekly pill for pediatric low-grade glioma, the most common childhood brain cancer. Now the company behind it, Day One Biopharmaceuticals, has agreed to be acquired by France's Servier for $2.5 billion in cash, a 68% premium over its previous closing share price.

Updated Mar 6

Reached PDUFA decision deadline on Sotyktu for psoriatic arthritis

For most of the past decade, people with psoriatic arthritis who wanted a pill instead of an injection had one real option: Janus kinase (JAK) inhibitors, a class of oral drugs that the Food and Drug Administration (FDA) slapped with its strongest safety warning in 2021 after a major trial linked them to higher rates of heart attacks, blood clots, cancer, and death. On March 6, the FDA reached its decision deadline on whether to approve the first oral drug from a different class entirely—Bristol Myers Squibb's Sotyktu (deucravacitinib), a selective tyrosine kinase 2 (TYK2) inhibitor—for adults with active psoriatic arthritis, a condition that causes painful joint inflammation in roughly 1.5 million Americans.

Updated Mar 6

Granted Breakthrough Therapy Designation to zorevunersen

For nearly five decades, children born with Dravet syndrome—a severe genetic epilepsy that causes hundreds of seizures per month, cognitive decline, and a mortality rate between 15 and 20 percent—had no treatment that addressed the underlying cause. Every available drug, including two approved in the past eight years, could only partially suppress symptoms. On March 5, 2026, the New England Journal of Medicine published the first clinical evidence that a drug can modify the disease itself: zorevunersen, an antisense oligonucleotide that boosts protein production from the healthy copy of the gene responsible for the condition, reduced motor seizures by up to 91 percent in 81 children and sustained improvements in cognition and behavior over three years.

Updated Mar 5

Developing framework for generative AI medical devices

The United States Food and Drug Administration (FDA) has cleared more than 1,250 artificial intelligence-enabled medical devices since it began tracking them — nearly all of them narrow systems that read scans or flag anomalies without ever speaking to a patient. On March 3, 2026, a startup called RecovryAI announced that the FDA granted Breakthrough Device Designation to its Virtual Care Assistants, making it the first patient-facing product built on a generative large language model (LLM) to enter the agency's accelerated review pipeline. The device is designed to be prescribed after joint replacement surgery, checking in with patients twice daily about sleep, activity, and diet, and escalating concerns to the surgical team.

Updated Mar 3

Approved three achondroplasia treatments: Voxzogo (daily), YUVIWEL (weekly), reviewing BridgeBio filing

For the estimated 250,000 people worldwide living with achondroplasia—the most common genetic cause of dwarfism—the only approved treatment required daily injections until BioMarin's Voxzogo. On February 12, 2026, BridgeBio reported that its oral pill achieved a 2.10 cm/year increase in height growth over placebo in a Phase 3 trial, exceeding Voxzogo's 1.57 cm/year gain and marking the first statistically significant improvement in body proportionality; two weeks later on February 27, the FDA approved Ascendis Pharma's once-weekly YUVIWEL (navepegritide) for children aged 2 and older.

Updated Mar 1

Has approved Dupixent nine times across distinct indications

When the Food and Drug Administration (FDA) approved Dupixent for severe eczema in 2017, it was one drug for one disease. Nine years later, the same molecule has been approved for nine separate conditions, from asthma to a chronic fungal sinus infection that previously had no approved treatment at all. The latest approval, granted on February 24, 2026, covers allergic fungal rhinosinusitis (AFRS), a condition that traps patients in a cycle of repeated sinus surgeries. In a pivotal trial, Dupixent reduced the need for additional surgeries and steroid courses by 92%.

Updated Feb 24

Issued draft guidance creating the plausible mechanism framework

For decades, the Food and Drug Administration (FDA) required the same basic proof for every drug: show it works in a controlled trial with enough patients to be statistically meaningful. That standard made sense for common diseases but created an impossible barrier for conditions affecting a handful of people worldwide. On February 23, 2026, the FDA issued draft guidance creating a fundamentally different standard—called the "plausible mechanism" framework—that would let developers of individualized gene-editing and ribonucleic acid (RNA) therapies win full approval by demonstrating their treatment targets the root genetic cause, successfully edits or engages the target, and improves outcomes compared to the disease's documented natural course.

Updated Feb 23

Continuing active review of psychiatric drug applications

Every atypical antipsychotic approved since the early 1990s has worked the same basic way: blocking dopamine receptors in the brain. In the past 17 months, the Food and Drug Administration (FDA) has approved three new psychiatric drugs—including Cobenfy, the first schizophrenia treatment with a genuinely novel mechanism in over 50 years, and now Bysanti, a new chemical entity from Vanda Pharmaceuticals cleared for both bipolar I disorder and schizophrenia. For the roughly 7 million Americans living with these conditions, the options just got meaningfully wider.

Updated Feb 21

Approved the venetoclax-acalabrutinib combination

For decades, patients diagnosed with chronic lymphocytic leukemia—the most common adult leukemia in Western countries, affecting roughly 23,000 Americans each year—faced a difficult choice: endure rounds of intravenous chemotherapy with harsh side effects, or take targeted pills indefinitely, sometimes for life. On February 20, 2026, the Food and Drug Administration (FDA) approved a combination of two oral drugs, venetoclax and acalabrutinib, that eliminates both burdens. Patients take pills for roughly one year, then stop. In a trial of 867 patients, 77% remained cancer-free at three years.

Updated Feb 20

Implementing three-pronged domestic manufacturing incentive strategy

Only 9% of the factories that make active pharmaceutical ingredients for American medicines are located in the United States. China and India account for roughly two-thirds of the rest. For decades, this arrangement kept drug prices low and went largely unchallenged — until the COVID-19 pandemic exposed how quickly a foreign export ban could empty American pharmacy shelves. Now the Food and Drug Administration (FDA) is quietly assembling what amounts to a three-layer incentive stack designed to reverse that dependency: the PreCheck pilot program to accelerate new factory buildouts, a priority review track for generics manufactured entirely on U.S. soil, and a proposed three-year fee waiver for new domestic plants under the next Generic Drug User Fee Amendments (GDUFA) reauthorization.

Updated Feb 20

Undergoing significant regulatory changes under new leadership

For 63 years, the Food and Drug Administration required drugmakers to prove their products worked in at least two rigorous clinical trials before Americans could take them. On February 18, 2026, Commissioner Marty Makary formally ended that standard, announcing that one trial will now be the "default position" for all new drugs—not just treatments for rare and fatal diseases, but medications for common conditions affecting millions of patients. In accompanying articles published in the New England Journal of Medicine and JAMA, Makary and Deputy Commissioner Vinay Prasad emphasized that the single-trial standard does not eliminate evidence requirements; instead, sponsors must provide "confirmative evidence" through mechanistic data, findings from related indications, animal models, real-world evidence, or data from drugs in the same class.

Updated Feb 20

Regulator of medical devices including bone grafts

For 24 years, Medtronic's INFUSE bone graft has followed a winding path through FDA approval, off-label controversy, lawsuits, and now expansion. The Food and Drug Administration (FDA) just granted INFUSE approval for transforaminal lumbar interbody fusion (TLIF) procedures—making it the only growth factor bone graft approved for two-level spinal constructs and the only one cleared for anterior, oblique, and transforaminal approaches.

Updated Feb 17

Supporting transition from animal testing to alternative models

For the first time, scientists have grown a miniature human spinal cord in a laboratory, injured it, and watched it heal. Northwestern University researchers published findings in Nature Biomedical Engineering showing their stem-cell-derived organoid accurately replicates cell death, inflammation, and scar formation seen in real spinal cord injuries—then demonstrated significant tissue repair when treated with an experimental therapy.

Updated Feb 16

Granted approval; previously designated Optune Pax as Breakthrough Device in December 2024

Pancreatic cancer has been oncology's grimmest frontier. The five-year survival rate sits at 13 percent—and for decades, patients diagnosed with locally advanced disease had essentially one option: chemotherapy that extended life by months, not years. On February 11, 2026, that changed. The Food and Drug Administration approved Optune Pax, a portable device that delivers alternating electrical fields to the abdomen, marking the first new FDA-approved treatment for locally advanced pancreatic cancer in nearly three decades.

Updated Feb 13

Adapting approval pathways for AI-driven precision medicine

For decades, cancer drug trials have failed at a rate exceeding 95%—burning through $50-60 billion annually on treatments tested in patients who were never likely to respond. On April 17, 2025, researchers from AstraZeneca and Tempus AI published results in Cancer Cell showing that the Predictive Biomarker Modeling Framework (PBMF)—a machine learning system using contrastive learning—can identify, from existing clinical data, which cancer patients will survive longer on immunotherapy versus chemotherapy. Applied retrospectively to completed phase 3 trials, the system improved survival outcomes by 15% compared to traditional patient selection.

Updated Feb 11

Evaluating rilzabrutinib under breakthrough therapy pathway

For 50,000 Americans living with warm autoimmune hemolytic anemia, there has never been a treatment specifically designed for their disease. On February 9, 2026, the Food and Drug Administration (FDA) granted breakthrough therapy designation to rilzabrutinib—marking the first time a Bruton's tyrosine kinase (BTK) inhibitor has reached this milestone for the condition. The designation signals the FDA's recognition that this drug may offer substantial improvement over existing therapies.

Updated Feb 9

Approved suzetrigine for moderate-to-severe acute pain

For more than 20 years, doctors facing patients in severe pain had two choices: prescribe opioids that risked addiction, or use weaker alternatives that often fell short. On January 30, 2025, the Food and Drug Administration (FDA) approved suzetrigine—brand name Journavx—ending that drought as the first entirely new class of pain medication since the late 1990s.

Updated Feb 7

Reviewing Anaphylm NDA with January 31, 2026 target date

For nearly four decades, Americans facing life-threatening allergic reactions had exactly one option: stab themselves with a needle. The EpiPen and its competitors delivered epinephrine effectively, but needle phobia kept roughly 30% of patients from using their prescribed devices during emergencies—contributing to preventable deaths. Now the needle era is ending. The Food and Drug Administration (FDA) approved the first needle-free alternative, a nasal spray called neffy, in August 2024. On February 2, 2026, the FDA issued a Complete Response Letter for Anaphylm, a dissolvable film placed under the tongue, rejecting the application but limiting deficiencies to human factors and packaging rather than safety or efficacy concerns. Aquestive Therapeutics expects to resubmit in the third quarter of 2026 after conducting a redesigned human factors validation study and a supporting pharmacokinetics study.

Updated Feb 5

Regulatory decision triggered underlying dispute

Delaware's courts have long served as the final word on American corporate disputes. On January 12, 2026, the state's Supreme Court issued a ruling that will reshape how companies structure billion-dollar acquisitions: when a merger agreement specifies a particular path to an earnout payment, courts will not rescue sellers who failed to negotiate alternatives.

Updated Feb 4

Granted breakthrough device designations and approved first non-invasive spinal cord stimulator

For decades, blood pressure instability has silently debilitated people with spinal cord injuries—78% of those with cervical injuries experience dangerous drops when sitting upright, yet only 28% receive treatment that actually works. ONWARD Medical just enrolled the first participant in a pivotal trial testing whether an implanted spinal cord stimulator can solve this problem by restoring the autonomic signals that paralysis disrupts.

Updated Feb 4

Approving new breast cancer treatments at accelerated pace

The U.S. breast cancer death rate has fallen 44% since its 1989 peak—an estimated 546,000 lives saved. Susan G. Komen's 2026 Progress Outlook attributes three-quarters of this decline to treatment advances and the remainder to earlier detection through mammography screening.

Updated Feb 3

Overseeing Neuralink's U.S. trials

Two years ago, Neuralink implanted a coin-sized chip in Noland Arbaugh's brain—the first human to receive the company's Telepathy device. As of January 28, 2026, twenty-one people across four countries are using Neuralink implants to control computers, phones, and robotic arms with their thoughts. Several participants now exceed the cursor-control speed of able-bodied people using a mouse.

Updated Feb 2

Developing cancer vaccine regulatory framework

Metastatic breast cancer typically kills most patients within five years. A small group of women vaccinated in a Duke University clinical trial two decades ago have defied that prognosis entirely—all remain alive today. Researchers discovered these survivors still carry specialized immune cells capable of recognizing their cancer, pointing to a mechanism that could make therapeutic cancer vaccines work reliably.

Updated Jan 31

Actively phasing out animal testing requirements in favor of organoid and AI-based methods

Johns Hopkins engineers grew miniature brains from patients' skin cells and discovered each psychiatric disorder has its own electrical fingerprint. The organoids diagnosed schizophrenia and bipolar disorder with 83% accuracy just by monitoring neural firing patterns—rising to 92% after gentle electrical stimulation. Machine learning algorithms spotted the differences invisible to human observers. The technology gained mainstream attention in January 2026 when NPR highlighted both the promise and ethical complexities of brain organoid research. By late January 2026, the American Psychiatric Association outlined plans to integrate biological biomarkers—including blood tests, neuroimaging, and digital monitoring—into the next DSM revision, signaling psychiatry's institutional shift toward biology-based diagnosis.

Updated Jan 31

Approved YUVEZZI as fourth presbyopia eye drop

For 240 years, the solution to presbyopia—the gradual loss of near-vision that affects nearly everyone over 45—has been essentially the same: put lenses in front of your eyes. Benjamin Franklin invented bifocals in 1785. Today, two billion people worldwide rely on reading glasses. On January 29, 2026, the Food and Drug Administration approved YUVEZZI (carbachol and brimonidine tartrate ophthalmic solution), the first dual-agent eye drop for treating this universal condition, marking a pivotal moment in pharmacological presbyopia treatment.

Updated Jan 30

Reviewing sparsentan sNDA with new April 13, 2026 PDUFA date

No FDA-approved treatment for focal segmental glomerulosclerosis (FSGS) has ever existed. For the 40,000 Americans with this rare kidney disease—which drives half of patients to kidney failure within a decade—the only options have been off-label immunosuppressants with mixed results. On January 13, 2026, that was supposed to change. Instead, the FDA extended its review of sparsentan by three months, requesting more data on clinical benefit. The announcement triggered a 33% intraday stock plunge and sparked multiple shareholder investigations into potential securities law violations. Two weeks later, CEO Eric Dube sold $1.56 million in company stock, a transaction drawing scrutiny given its timing.

Updated Jan 30

Granted Breakthrough Therapy Designation

Cancer vaccines have promised to train the immune system against tumors for decades. None has delivered a durable, replicable benefit—until now. On January 20, 2026, Moderna and Merck reported that their personalized mRNA vaccine, combined with the immunotherapy Keytruda, cut the risk of melanoma recurrence or death by 49% at five years in a Phase 2b trial of 157 patients. The sustained result—identical to the three-year mark—suggests the vaccine permanently reprograms immune surveillance rather than offering temporary protection.

Updated Jan 25

Approved generic CF drugs after patent expiration

A generic version of Trikafta—the breakthrough cystic fibrosis drug that extends lives by decades—hit the market at $12,750 per year. That's 95% cheaper than Vertex Pharmaceuticals' brand-name version, which costs over $300,000 annually and has generated billions while remaining out of reach for patients in developing nations and many Americans with inadequate insurance.

Updated Jan 12

Approved six CAR-T therapies; removed REMS requirements in 2025

Gene therapy killed Jesse Gelsinger in 1999. His death triggered FDA shutdowns, lawsuits, and nearly ended the field. Fast forward to December 2025: British scientists published results showing that base-edited immune cells—a 'living drug'—achieved deep remissions in 82% of children with previously incurable T-cell leukemia. Three years after treatment, 64% remain disease-free. Some patients are off treatment entirely.

Updated Jan 9

Developing joint CDER-CBER guidance on plausible mechanism pathway

In August 2024, KJ Muldoon was born with a death sentence encoded in his DNA—a single broken letter among three billion that left his body unable to process protein. Six months later, he walked out of Children's Hospital of Philadelphia, cured by a gene-editing therapy that didn't exist when he was diagnosed. The treatment was designed, manufactured, approved, and delivered in 180 days.

Updated Jan 7