Federal agency
Appears in 78 stories
Regulator reviewing DF-003's path to market
A few dozen families worldwide carry a gene mutation that slowly steals their sight and floods their bodies with inflammation. There is no approved drug that treats the cause. On July 13, 2026, Drug Farm said it raised $55 million to move its candidate for that disease, ROSAH syndrome, toward a Phase 3 trial.
Updated Yesterday
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 found these survivors still carry specialized immune cells that recognize their cancer, offering a model for why some therapeutic vaccines produce lasting results.
Updated 5 days ago
Approved six CAR-T therapies; removed REMS requirements in 2025
Gene therapy killed Jesse Gelsinger in 1999. His death triggered FDA shutdowns and nearly ended the field.
Updated Jul 3
Regulator that granted the approval
More than 8 in 10 donor lungs recovered from deceased donors never reach a patient. On June 29, 2026, the FDA approved a device built to change that math. United Therapeutics won premarket approval for LungFX, a system that keeps donor lungs warm, ventilated, and pumped with fluid outside the body so transplant teams can re-examine organs they had already rejected.
Updated Jun 29
Granted both first-line approvals
Triple-negative breast cancer is aggressive and short on options. On June 24, 2026, the U.S. Food and Drug Administration cleared Gilead Sciences' Trodelvy as a first-line treatment, moving a drug once used only after other therapies failed to the front of the line. Six days earlier, the European Commission had done the same for patients in the EU who are ineligible for immunotherapy.
Updated Jun 25
Primary regulator of AI medical devices in the United States
University of Michigan researchers published Prima in February 2026, an AI that reads brain MRIs in seconds with up to 97.5% accuracy across 50 neurological conditions. In April 2026, Harvard Medical School reported that OpenAI's o1 model correctly diagnosed emergency patients 67% of the time, compared to 50–55% for two attending physicians reviewing the same cases.
Updated Jun 24
Approved Colorado's program; oversees state imports
On June 15, 2026, the FDA approved Colorado's plan to import about 20 prescription medicines from Canada. The state estimates residents will save 20% to 70% on those drugs, totaling roughly $46 million over three years.
Updated Jun 16
Granted the approval
Surgery removes the tumor. For many high-risk kidney cancer patients, it doesn't end the disease. On June 12, 2026, the U.S. Food and Drug Administration (FDA) approved pairing belzutifan with the immunotherapy pembrolizumab to keep these patients cancer-free for longer after their kidney is removed.
Updated Jun 12
Reviewing darovasertib through its Real-time Oncology Review program
IDEAYA Biosciences closed a $323.6 million stock sale on June 10, 2026. The cash lands as the company prepares to file its first drug with the U.S. Food and Drug Administration (FDA).
Updated Jun 10
Reviewing both drugs
GSK agreed to pay $10.6 billion in cash for Nuvalent, a Cambridge, Massachusetts biotech with two lung cancer drugs awaiting U.S. approval. At $124 a share, the price is about 40% above where Nuvalent traded before the deal. It is GSK's biggest acquisition in more than a decade.
Updated Jun 9
Granted Breakthrough Therapy Designation
Some children with spinal muscular atrophy get a one-time gene therapy, then slowly weaken again. There has been no clear next step for them. On June 4, 2026, the FDA gave Biogen's salanersen a designation meant to speed up that next step.
Updated Jun 4
Granted approval; previously designated Optune Pax as Breakthrough Device in December 2024
Pancreatic cancer kills roughly 52,000 Americans each year and carries a five-year survival rate of 13 percent. In February 2026, the FDA approved Optune Pax, the first new treatment for locally advanced disease in nearly 30 years, adding alternating electric fields to the treatment options alongside chemotherapy.
Updated Jun 3
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 May 31
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.
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 FDA approved Bizengri (zenocutuzumab-zbco) from Partner Therapeutics as the first drug targeting this NRG1 fusion, blocking the abnormal protein it produces, which fuels tumor growth.
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.
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 FDA extended ADMA's ASCENIV to children as young as two, ending the reliance on off-label or alternatives.
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, Mirum Pharmaceuticals reported that its experimental pill volixibat hit the primary endpoint of its Phase 2b VISTAS trial, reducing the relentless itch far more than placebo.
Approved first-in-class PROTAC 35 days early
For 25 years, drug companies have tried to design medicines that destroy harmful proteins outright rather than blocking them. On May 1, the U.S. Food and Drug Administration (FDA) approved the first one.
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 from the University of Barcelona and Oregon Health & Science University reported that engineered DNA reduced LDL by nearly 50 percent in mice. A single injection of this engineered DNA molecule silences the gene controlling how the liver clears LDL.
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 FDA approved Axsome Therapeutics' Auvelity for the indication, doubling approved options from one to two. The FDA called it the 'first non-antipsychotic drug to treat agitation associated with dementia' — what sets it apart from Rexulti.
Receiving rolling BLA submission for lonvo-z under RMAT designation; full submission expected H2 2026
On April 27, 2026, Intellia Therapeutics reported results from Phase 3 testing of lonvoguran ziclumeran (lonvo-z), a one-time in-body CRISPR gene-editing therapy for hereditary angioedema. The 80-patient randomized double-blind trial found a single IV infusion cut debilitating swelling attack rates by 87% versus placebo (weeks 5-28); 62% of patients became attack-free and stopped all preventive medications. The safety profile was clean: no serious adverse events, and infusion-related reactions, headache, and fatigue—the most common complaints—were mild or moderate.
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 — it cuts that timeline to as little as two months. They simultaneously paused the existing Transitional Coverage for Emerging Technologies (TCET) program for new applicants, consolidating all breakthrough-device coverage work under RAPID.
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. On April 18, 2026, President Trump signed an executive order 'Accelerating Medical Treatments for Serious Mental Illness' directing the FDA to expedite clinical trials of those substances for PTSD in veterans.
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 bridges the patient's immune cells to their tumor has won approval in both the United States and China.
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.
Updated May 30
Approved Foundayo under new fast-track voucher program
For two years, the most effective weight-loss drugs on the market required a weekly injection, but that barrier is now falling. Eli Lilly began shipping Foundayo on April 6, 2026 — just five days after FDA approval in a record 50-day review — making it the first oral GLP-1 pill with no food or water restrictions. At $149/month (or $25 with insurance), it's 85% cheaper than injectables.
Granted RMAT designation to CB-011
Every approved cancer cell therapy today requires extracting a patient's own cells, engineering them in a lab, and infusing them back. It costs over $400,000 and sometimes takes weeks — long enough that patients' cancer can grow while they wait.
Reviewing relutrigine NDA under priority review
Children with SCN2A and SCN8A developmental and epileptic encephalopathies have never had a drug designed for their condition, cycling through four or more unapproved medications as seizures persist and development regresses. The FDA accepted Praxis Precision Medicines' application for relutrigine and granted priority review, with a decision expected by September 27, 2026.
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 FDA approved Awiqli, a once-weekly basal insulin from Novo Nordisk for adults with type 2 diabetes. That cuts basal insulin injections from 365 to 52 per year.
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.
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 FDA approved Denali Therapeutics' AVLAYAH. It's the first drug specifically engineered to cross the blood-brain barrier and deliver a therapeutic enzyme to the central nervous system after a simple intravenous infusion.
Granted expanded indication for OmniaSecure
For decades, defibrillators have saved lives by shocking dangerously fast rhythms back to normal, but the leads also pace the heart unnaturally and can cause damage over time. Medtronic's OmniaSecure lead is the first defibrillation lead that can pace through the heart's own electrical wiring, closely mimicking natural heartbeats. It has been cleared by the FDA for placement in the left bundle branch area.
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 9,000+ participants. They will seek FDA approval this year for a possible 2027 launch.
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 semaglutide (the compound in Ozempic and Wegovy) reduced psychiatric hospitalizations, worsening depression, and substance-use crises.
Approves fourth obesity drug in 13 months: Imcivree (rare), high-dose Wegovy, oral semaglutide, and Foundayo (common)
For the first time, patients with acquired hypothalamic obesity have an FDA-approved treatment. On March 19, 2026, the FDA approved Imcivree (setmelanotide), which targets this rare condition affecting roughly 10,000 Americans whose hypothalamic damage from brain tumor surgery makes diet and exercise ineffective.
Approved ICOTYDE for moderate-to-severe plaque psoriasis
For two decades, treating moderate-to-severe psoriasis required needles — self-injected drugs or clinic infusions many patients refused or abandoned. On March 18, 2026, the FDA approved ICOTYDE, a once-daily pill that blocks the IL-23 receptor like injectable biologics. In 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 placebo.
Reviewing antibiotic candidates under expedited pathways
No genuinely new class of antibiotic has reached patients since 1987, and in the nearly four decades since, bacteria have steadily evolved resistance to existing drugs. Carbapenem-resistant Acinetobacter baumannii—a hospital-acquired pathogen that kills up to 60 percent of ventilated pneumonia patients—is at the top of the WHO's list of critical-priority threats. On March 16, 2026, Swiss biotech BioVersys received FDA clearance to enroll American patients in a Phase 3 pivotal trial of BV100, which reduced 28-day mortality by half in earlier testing.
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 vision range, but dimmer, lower-contrast sight—especially in low light. On March 12, 2026, the FDA approved Johnson & Johnson's TECNIS PureSee, the first extended depth of focus lens cleared in the U.S. without a contrast sensitivity warning.
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.
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 FDA cleared Ojemda, a once-weekly pill for pediatric low-grade glioma, the most common childhood brain cancer. Day One Biopharmaceuticals, the company behind it, has agreed to be acquired by France's Servier for $2.5 billion in cash—a 68% premium over its previous closing share price.
Reached PDUFA decision deadline on Sotyktu for psoriatic arthritis
For most of the past decade, people with psoriatic arthritis who wanted a pill had one real choice: Janus kinase (JAK) inhibitors. The FDA slapped those drugs with its strongest safety warning in 2021, after a major trial linked them to higher rates of heart attacks, blood clots, cancer, and death.
Granted Breakthrough Therapy Designation to zorevunersen
Dravet syndrome is a severe genetic epilepsy that causes hundreds of seizures per month, cognitive decline, and a 15–20 percent mortality rate. For nearly five decades, no treatment addressed the underlying cause — including two drugs approved in the past eight years, which could only partially suppress symptoms.
Developing framework for generative AI medical devices
The FDA has cleared more than 1,250 artificial intelligence-enabled medical devices — nearly all narrow systems that read scans or flag anomalies without talking to patients. On March 3, 2026, RecovryAI announced that its Virtual Care Assistants were granted Breakthrough Device Designation, the first patient-facing generative large language model product to enter the FDA's accelerated review pipeline.
Has approved Dupixent nine times across distinct indications
Dupixent was approved for severe eczema in 2017 as a one-disease drug. Nine years later, it's approved for nine conditions, with the latest approval (February 24, 2026) covering allergic fungal rhinosinusitis (AFRS) — which previously had no approved treatment — and reducing the need for additional surgeries and steroid courses by 92% in trials.
Updated May 29
Issued draft guidance creating the plausible mechanism framework
For decades, the FDA required all drugs to prove effectiveness through controlled trials with sufficient sample sizes, a standard that made sense for common diseases but created an impossible barrier for ultra-rare conditions. On February 23, 2026, the FDA issued draft guidance for a new standard—the 'plausible mechanism' framework. Developers of gene-editing and ribonucleic acid (RNA) therapies could win full approval by showing that their treatment targets the root genetic cause, engages it, and improves outcomes compared to the disease's natural course.
Continuing active review of psychiatric drug applications
The FDA approved Cobenfy for schizophrenia in September 2024—the first drug to treat the condition through muscarinic receptors rather than dopamine, a different approach than any approved in the previous 50 years. Bysanti (milsaperidone) followed in February 2026, cleared for both schizophrenia and bipolar I disorder. For the roughly 7 million Americans with these conditions, the treatment menu is wider than it was two years ago.
Approved the venetoclax-acalabrutinib combination
On February 20, 2026, the FDA approved venetoclax and acalabrutinib for chronic lymphocytic leukemia (CLL), the most common adult leukemia in Western countries, with roughly 23,000 new American cases each year. In a trial of 867 patients, 77% remained cancer-free at three years.
Implementing three-pronged domestic manufacturing incentive strategy
Only 9% of the factories making active pharmaceutical ingredients for American medicines are in the U.S.; China and India account for roughly two-thirds. 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. The FDA is assembling three programs to reverse this dependency: PreCheck to accelerate new factory buildouts, priority reviews for domestically made generics, and a proposed three-year fee waiver for new domestic plants under the next Generic Drug User Fee Amendments (GDUFA) reauthorization.
Undergoing significant regulatory changes under new leadership
For 63 years, the FDA required drugmakers to prove their products worked in at least two rigorous clinical trials. On February 18, 2026, Commissioner Marty Makary formally ended that standard. Now one trial is the 'default position' for all new drugs—from rare diseases to 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 doesn't 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.
Regulator of medical devices including bone grafts
Medtronic's INFUSE bone graft has spent 24 years navigating FDA approval, off-label controversy, and lawsuits. The FDA just approved INFUSE for transforaminal lumbar interbody fusion (TLIF) procedures—the only growth factor bone graft approved for two-level spinal constructs and the only one cleared for anterior, oblique, and transforaminal approaches.
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 organoid replicates cell death, inflammation, and scarring in spinal cord injuries and shows tissue repair when treated with an experimental therapy.
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.
Approved three achondroplasia treatments: Voxzogo (daily), YUVIWEL (weekly), reviewing BridgeBio filing
About 250,000 people worldwide have achondroplasia, the most common genetic cause of dwarfism. Until BioMarin's Voxzogo, the only approved treatment required daily injections.
January 2026 wellness guidance allows blood pressure estimation without clearance; cardiologists publicly raising accuracy concerns
Oura's fifth-generation smart ring went on sale Thursday with blood pressure monitoring and on-demand telehealth access, starting at $399. The Ring 5 is 40% smaller than its predecessor and monitors nighttime blood pressure shifts that can warn of cardiovascular strain.
Approving agency for Decnupaz
The FDA approved AbbVie's Decnupaz on May 27 for blastic plasmacytoid dendritic cell neoplasm, an aggressive blood cancer. It strikes an estimated few hundred Americans a year. It's the second drug ever approved for the disease and the first doctors can start in an outpatient clinic.
Updated May 28
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 unlikely to respond. On April 17, 2025, researchers from AstraZeneca and Tempus AI published in Cancer Cell results from the Predictive Biomarker Modeling Framework, a machine learning system using contrastive learning on existing clinical data. The system can identify which cancer patients will survive longer on immunotherapy versus chemotherapy. Applied retrospectively to completed phase 3 trials, it improved survival outcomes by 15% compared to traditional patient selection.
Updated May 27
Accepted Helix pre-submission for substantive review; Q2 2026 meeting scheduled; reviewing CardiAMP accelerated approval request
In March 2026, the FDA accepted BioCardia's pre-submission package for the Helix Transendocardial Delivery Catheter, the first catheter that injects cell and gene therapies directly into heart muscle, and scheduled a formal meeting for Q2 2026. The device, backed by fifteen clinical trials, uses a small helical needle anchored within the beating heart to precisely deliver therapeutic agents to damaged tissue; CDRH leads the review in consultation with CBER.
Evaluating rilzabrutinib under breakthrough therapy pathway
For 50,000 Americans with warm autoimmune hemolytic anemia, there's never been a disease-specific treatment. On February 9, 2026, the FDA granted breakthrough therapy designation to rilzabrutinib—the first BTK inhibitor to reach this milestone for the condition—signaling it may offer substantial improvement over existing therapies.
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.
Granted breakthrough device designations and approved first non-invasive spinal cord stimulator
Blood pressure instability has long burdened people with spinal cord injuries. Among those with cervical injuries, 78% experience dangerous drops when sitting upright, yet only 28% receive effective treatment. ONWARD Medical just enrolled the first participant in a trial testing whether an implanted spinal cord stimulator can restore the autonomic signals that paralysis disrupts.
Updated May 26
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.
Regulatory decision triggered underlying dispute
Delaware's courts rule on American corporate disputes. On January 12, 2026, the state's Supreme Court decided that when a merger agreement specifies a particular path to an earnout payment, courts won't rescue sellers who failed to negotiate alternatives.
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 use Neuralink implants to control computers, phones, and robotic arms with their thoughts; several now exceed the cursor-control speed of able-bodied people using a mouse.
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.
Updated May 23
Approved YUVEZZI as fourth presbyopia eye drop
For 240 years, reading glasses have treated presbyopia—the gradual loss of near vision that affects nearly everyone over 45—unchanged since Benjamin Franklin invented bifocals in 1785. Two billion people worldwide still rely on reading glasses today. On January 29, 2026, the FDA approved YUVEZZI (carbachol and brimonidine tartrate ophthalmic solution), the first dual-agent eye drop for presbyopia.
Approved Yartemlea after extended review process
For decades, patients with transplant-associated thrombotic microangiopathy (TA-TMA) after stem cell transplants faced no FDA-approved treatment—mortality rates reached 80-90% for severe cases. In December 2025, the FDA approved Yartemlea, the first therapy for TA-TMA.
Cancer vaccines have promised to train the immune system against tumors for decades. None has delivered a durable, replicable benefit until now.
Updated May 22
Reviewing NXC-201; will rule on eventual Biologics License Application
Immix Biopharma reported Thursday that 19 of 20 patients in its NEXICART-2 Phase 2 trial reached complete remission with NXC-201, a one-time CAR-T cell therapy for AL amyloidosis. The 95% complete response rate is up from 75% in an earlier readout, and all four patients with minimal-residual-disease-negative status have now converted to complete responses.
Updated May 21
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 to kidney failure within a decade—the only options have been off-label immunosuppressants with mixed results.
Approved generic CF drugs after patent expiration
A generic version of Trikafta—the 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. The brand-name drug has generated billions while remaining out of reach for patients in developing nations and many Americans with inadequate insurance.
Updated May 20
Approved Guardant360 Liquid CDx on May 20, 2026
For most patients with advanced cancer, picking the right targeted drug still starts with a tissue biopsy. On May 20, the FDA approved a blood test that reads 100 times more of the tumor's DNA than the previous version.
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 designed, manufactured, approved, and delivered in 180 days.
Updated May 19
Granted Breakthrough Therapy Designation to TERN-701
Merck borrowed billions in the investment-grade bond market on Monday. The cash refinances its $6.7 billion all-cash purchase of Terns Pharmaceuticals, a small California biotech the company closed on two weeks ago.
Updated May 18
Approved both new indications; also cleared companion diagnostic tests
The U.S. Food and Drug Administration (FDA) cleared AstraZeneca and Daiichi Sankyo's Enhertu for two new uses in early-stage HER2-positive breast cancer in May 2026. The drug, already a standard for advanced disease, now enters the window where the goal is cure, not control.
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.
Updated May 16
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 May 15
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 May 14 stay is all that blocks a May 1 Fifth Circuit ruling ending telehealth mifepristone prescribing and mail delivery nationwide.
Updated May 13
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