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PTC Therapeutics withdraws Translarna NDA

USA-based rare disease drug developer PTC Therapeutics saw its shares drop 5.2% to $67.05 after market close, when it announced that it has withdrawn the New Drug Application (NDA) resubmission for Translarna (ataluren) for the treatment of nonsense mutation Duchenne muscular dystrophy (DMD) following US Food and Drug Administration (FDA) feedback on the application review. 13 February 2026

FDA accepts Takeda’s oveporexton NDA for priority review

The US Food and Drug Administration (FDA) has accepted Takeda’s New Drug Application (NDA) and granted Priority Review for oveporexton (TAK-861) for the treatment of narcolepsy type 1 (NT1). 10 February 2026

Sanofi’s rilzabrutinib wins ‘Breakthrough’ status from FDA for wAIHA

The US Food and Drug Administration (FDA) has granted a designation as breakthrough therapy to Wayrilz (rilzabrutinib), a novel oral, reversible Bruton’s tyrosine kinase (BTK) inhibitor, for the treatment of patients with warm autoimmune hemolytic anemia (wAIHA), a rare autoimmune disorder marked by the destruction of red blood cells, French pharma major Sanofi announced this morning. 9 February 2026

FDA to take action against non-FDA-approved GLP-1 drugs

US Department of Health and Human Services general counsel Mike Stuart announced Friday that his office referred Hims & Hers Health. to the US Department of Justice for investigation, a day after Novo Nordisk threatened litigation over what it called the telehealth company's "knockoff" version of its popular weight loss drug Wegovy (semaglutide). 9 February 2026

China’s NMPA talks global policy and strategy with FDA

Yang Sheng, deputy commissioner of the National Medical Products Administration (NMPA) of China, met with a visiting delegation led by Mark Abdoo, associate commissioner for global regulatory operations and policy of the US Food and Drug Administration (FDA). 9 February 2026

The week in pharma: action, reaction and insight – week to February 6, 2026

Last week’s regulatory news kicked of with a batch of Food and Drug Administration (FDA) completes response letters (CRLs), including for Aquestive Therapeutics’ Anaphylm and AstraZeneca’s Saphnelo for systemic lupus erythematosus. Eli Lilly and Novo Nordisk, the leaders and rivals in the weight management space – with their respective Mounjaro/Zepbound and Wegovy brands - both released financial results and updates that attracted attention. 8 February 2026

FDA grants priority review for Hympavzi sBLA

Pfizer today announced that the US Food and Drug Administration (FDA) has accepted and granted Priority Review for the company’s supplemental Biologics License Application (sBLA) for Hympavzi (marstacimab) to expand the approved indication to include the treatment of hemophilia A or B patients 6 years and older with inhibitors, and pediatric patients (ages 6 to 11) with hemophilia A or B without inhibitors. 6 February 2026

Relay Thera’s zovegalisib wins ‘Breakthrough’ status from FDA

Cambridge, USA-based precision medicines specialist Relay Therapeutics today announced that the US Food and Drug Administration (FDA) has granted Breakthrough Therapy designation (BTD) to zovegalisib (RLY-2608). 3 February 2026

FDA issues CRL on AstraZeneca’s Saphnelo BLA

The US Food and Drug Administration (FDA) has issued a complete response letter (CRL) regarding the Biologics License Application (BLA) for Saphnelo (anifrolumab) for subcutaneous administration in adult patients with systemic lupus erythematosus (SLE). 3 February 2026

A look at new medicines approved in 2025 in USA

This past year, innovative biopharmaceutical companies received approval for 52 new medicines - 46 new drugs and 6 new biologics and vaccines - demonstrating the industry’s commitment to advancing health for patients across a wide range of diseases. 3 February 2026

Aquestive leaps, despite receiving CRL for Anaphylm NDA

Shares of US pharma Aquestive Therapeutics rocketed 45.4% to $4.20 this morning, despite receiving a Complete Response Letter (CRL) from the US Food and Drug Administration (FDA) on January 30 for its New Drug Application (NDA) seeking approval of Anaphylm (dibutepinephrine). 2 February 2026

FDA slaps CRL on Pharming’s sNDA for Joenja

Dutch biopharma company Pharming Group saw its shares tumble 13.5% to 1.48 euros this morning after it received a regulatory setback. 2 February 2026

US regulator accepts Summit filing for ivonescimab combo

The US Food and Drug Administration has accepted Summit Therapeutics’ application for ivonescimab plus chemotherapy in patients with EGFR-mutated non-small-cell lung cancer who have progressed after tyrosine kinase inhibitor treatment. A decision is due by mid-November. 30 January 2026

FDA approves Tenpoint’s Yuvezzi

The US Food and Drug Administration (FDA) has approved Yuvezzi (carbachol and brimonidine tartrate ophthalmic solution) 2.75%/0.1%, previously known as Brimochol PF, from UK-based ophthalmics specialist Tenpoint Therapeutics 29 January 2026

Regenxbio shares hit by setback with gene therapies

The US Food and Drug Administration (FDA) has placed a clinical hold on Regenxbio’s investigational gene therapy, RGX-111, for the treatment of MPS I, also known as Hurler syndrome, following the development of a brain tumor in a participant treated in its Phase I/II study. 28 January 2026

FDA ‘Breakthrough’ status for Biogen’s litifilimab

The US Food and Drug Administration (FDA) has granted Breakthrough Therapy designation for litifilimab (BIIB059) for the treatment of cutaneous lupus erythematosus (CLE), the drug’s developer, US biotech major Biogen, announced today. 28 January 2026

FDA approves new indication for Darzalex Faspro combo

The US Food and Drug Administration (FDA) yesterday approved daratumumab and hyaluronidase-fihj – trade name Darzalex Faspro - from Johnson & Johnson subsidiary Janssen Biotech, in combination with bortezomib, lenalidomide, and dexamethasone (VRd) for adults with newly diagnosed multiple myeloma who are ineligible for autologous stem cell transplant (ASCT). 28 January 2026

FDA accepts Otsuka’s centanafadine NDA for priority review

The Food and Drug Administration (FDA) has accepted for priority review Japanese drugmaker Otsuka’s (TYO: 4578) New Drug Application (NDA) for centanafadine, an investigational, once-daily extended release capsule and the first-in-class norepinephrine, dopamine, and serotonin reuptake inhibitor (NDSRI. 27 January 2026

Intellia shares leap as FDA lifts trial hold

Gene editing company Intellia Therapeutics has announced that the US Food and Drug Administration (FDA) has removed the clinical hold on the Investigational New Drug application (IND) for the MAGNITUDE-2 Phase III trial of nexiguran ziclumeran (nex-z) for patients with hereditary transthyretin amyloidosis with polyneuropathy (ATTRv-PN). 27 January 2026

FDA accepts Leqembi sBLA for at-home injection under priority review

Japan’s Eisai and US biotech Biogen have announced that the US Food and Drug Administration (FDA) has accepted for review the former’s Supplemental Biologics License Application (sBLA) for lecanemab-irmb subcutaneous autoinjector (SC-AI), branded as Leqembi Iqlik, as a weekly starting dose. 26 January 2026

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FDA draft guidance on genome editing safety standards

The US Food and Drug Administration (FDA) today issued a draft guidance for sponsors seeking approval of human gene therapy products involving genome editing technologies. 14 April 2026

Travere’s Filspari wins landmark US approval

US biopharma Travere Therapeutics has announced that the US Food and Drug Administration (FDA) has approved Filspari (sparsentan) to reduce proteinuria in adult and pediatric patients aged eight years and older with focal segmental glomerulosclerosis (FSGS) without nephrotic syndrome. 14 April 2026

SynOx Therapeutics heading for FDA filing for emactuzumab in TGCT

Anglo-Irish biopharma SynOx Therapeutics announced that the US Food and Drug Administration (FDA) has granted Fast Track designation (FTD) to emactuzumab for the treatment of tenosynovial giant cell tumors (TGCT) patients that are not amenable to or who would not benefit from surgery. 14 April 2026

The week in pharma: action, reaction and insight – week to April 10, 2026

Important news last week kicked off with Neurocrine Biosciences announcing a proposed $2.9 billion takeover bid for Solena Therapeutics. Then Gilead Sciences revealed an up to $5 billion takeover for German antibody-drug conjugate (ADCs) developer Tubulis. Also of note, Sanofi released mixed Phase II results for its asthma and dermatology candidate lunsekimig. The US Food and Drug Administration (FDA) announced plans for greater control of direct-to-consumer Rx medicines advertising. 12 April 2026

Annovis secures financing for buntanetap’s NDA submission

USA-based neurodegenerative disorders specialist Annovis Bio today revealed that it has secured financing to support the development of its investigational oral therapy, buntanetap, for neurodegenerative diseases such as Alzheimer’s and Parkinson’s. 10 April 2026

Addressing DTC advertising among FDA priorities

The US Food and Drug Administration (FDA) has outlined a number of legislative proposals in its fiscal 2027 budget request. 8 April 2026

FDA approves first generic dapagliflozin tablets

The US Food and Drug Administration approved the first generics of UK pharma major AstraZeneca’s Farxiga (dapagliflozin) tablets to reduce the risk of hospitalization for heart failure in adults with type 2 diabetes mellitus and either established cardiovascular disease or multiple cardiovascular risk factors, and as an adjunct to diet and exercise to improve glycemic control in adults with type 2 diabetes mellitus. 8 April 2026

FDA-approved drug manufacturing deals shift to Europe

In 2025, US contract manufacturing deals for US Food and Drug Administration (FDA)-approved drugs posted their steepest decline in five years. Despite record-high US tariffs on EU pharmaceuticals, biopharma companies, including US-based firms, are increasingly choosing to outsource manufacturing for the US market to facilities in Europe rather than shifting work to domestic providers. 7 April 2026

Regulatory round-up: 7 April 2026

The past week produced a thinner regulatory crop than some recent weeks, but there were still several reportable human-medicines developments across major agencies. The clearest headline came from the US Food and Drug Administration, which approved Foundayo (orforglipron) under its National Priority Voucher program. 7 April 2026

The week in pharma: action, reaction and insight – week to April 3, 2026

Important research news last week included US pharma giant Merck & Co moving its Winrevair into Phase III for pulmonary arterial hypertension. On the M&A front, Biogen announced a $5.6 billion takeover for Apellis and its two main products – Empaveli and Syfovre. Eli Lilly’s oral obesity drug orforglipron has been granted US Food and Drug Administration (FDA) in a record time, under the trade name Foundayo. Also of note, Sino-American biotech Adagene struck a deal with the USA’s Incyte to evaluate muzastotug in colorectal cancer. 5 April 2026

FDA review not plain sailing for Orca

Privately-held US cell therapy company Orca Bio announced that the US Food and Drug Administration (FDA) has extended the review timeline of its Biologics License Application (BLA) for Orca-T for the treatment of patients with hematologic malignancies. 2 April 2026

Fore Biotherapeutics wins ‘Breakthrough’ status for plixorafenib

Precision oncology start-up Fore Biotherapeutics announced that the US Food and Drug Administration (FDA) has granted Breakthrough Therapy designation (BTD) to plixorafenib for the treatment of adult patients with BRAF V600E-mutated high-grade glioma (HGG). 2 April 2026

US FDA nod for label extensions of Alyftrek and Trikafta

US drugmaker Vertex Pharmaceuticals today announced the US Food and Drug Administration (FDA) has approved expanded indications for two of its top-selling drugs. 1 April 2026

FDA identifies cases of serious liver injury in patients taking Tavneos

The US Food and Drug Administration (FDA) is alerting patients and health care professionals about serious post-marketing cases, including fatal cases, of drug-induced liver injury (DILI) associated with US biotech major Amgen’s Tavneos (avacopan). 1 April 2026

Teva gains biosimilars momentum with FDA approvals

Israel-based Teva Pharmaceutical Industries today announced three milestones in its biosimilar portfolio, demonstrating continued momentum in the advancement of its Pivot to Growth strategy. 30 March 2026

March 2026: Pharmacological advancements in rare diseases

March 2026 marked a significant inflection point for rare disease therapeutics, as regulatory momentum, policy pressure, and scientific innovation converged. 30 March 2026

FDA approves Rocket Pharma’s Kresladi for severe LAD-I

The US Food and Drug Administration (FDA) has granted accelerated approval to Kresladi (marnetegragene autotemcel) for the treatment of pediatric patients with severe leukocyte adhesion deficiency-I (LAD-I), US biotech Rocket Pharmaceuticals announced today. 27 March 2026

Novo Nordisk’s Awiqli approved by FDA

Danish pharma major Novo Nordisk announced that the US Food and Drug Administration (FDA) has approved Awiqli (insulin icodec-abae) injection 700 units/mL, the first and only once-weekly, long-acting basal insulin, indicated as an adjunct to diet and exercise to improve glycemic control (blood sugar) in adults living with type 2 diabetes. 27 March 2026

FDA publishes 2025 GDUFA science and research report

The US Food and Drug Administration FDA’s Center for Drug Evaluation and Research published its fiscal year 2025 Generic Drug User Fee Amendments (GDUFA) Science and Research Report at https://www.fda.gov/drugs/generic-drugs/fy-2025-gdufa-science-and-research-report. 27 March 2026

FDA orders warning for Parkinson’s mainstay after seizure cases

The US Food and Drug Administration is requiring new label warnings for widely used Parkinson’s therapies combining levodopa and carbidopa, citing a risk of vitamin B6 deficiency that may trigger seizures. 26 March 2026

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