Biologics
Sarepta Therapeutics is announcing that its gene therapy Elevidys significantly slowed disease progression over three years in ambulatory patients with Duchenne muscular dystrophy, according to topline results from the Phase 3 EMBARK trial. The company says patients treated with Elevidys maintained higher levels of motor function than untreated controls across
Biologics
A coordinated advocacy campaign by the Huntington’s disease community helped set the stage for a high-priority meeting between FDA and uniQure to discuss the regulatory path forward for the company’s experimental gene therapy AMT-130. UniQure said earlier this month that it had secured a Type A
Biologics
Moderna and Merck say that updated five-year follow-up data show that their personalized mRNA cancer vaccine candidate, intismeran autogene, significantly reduced the risk of melanoma recurrence or death when used in combination with Merck’s immunotherapy Keytruda (pembrolizumab), compared with Keytruda alone, according to the companies. In a
Biologics
FDA has asked Capricor Therapeutics for a full clinical study report from the company’s Phase 3 HOPE-3 trial as part of its ongoing BLA review for Deramiocel, a proposed cell therapy for Duchenne muscular dystrophy. The request follows the agency’s review of topline HOPE-3 data that
Biologics
Valneva says it has voluntarily withdrawn its BLA for Ixchiq, the company’s chikungunya vaccine, based on FDA safety-related restrictions on the product. In 8/2025, CBER suspended Valneva’s BLA due to serious safety concerns that the vaccine could be causing chikungunya-like illness in vaccine recipients. The
Biologics
Scholar Rock says it is planning to resubmit this year its BLA seeking approval of apitegromab, a first-in-class muscle-targeted therapy for spinal muscular atrophy (SMA). In 9/2025, FDA issued a complete response letter that cited observations made during an FDA inspection of Catalent Indiana, a third-
Biologics
FDA has accepted for priority review an Argenx supplemental BLA seeking to expand the use of its myasthenia gravis drug Vyvgart (efgartigimod alfa-fcab) to adults with acetylcholine receptor antibody–negative generalized myasthenia gravis. The agency set a user fee review action target date of 5/10. If approved, the
Biologics
FDA has accepted for review a PharmaEssentia supplemental BLA seeking to expand the label of its interferon therapy Besremi (ropeginterferon alfa-2b-njft) to include adults with essential thrombocythemia. The agency has set a user fee review action target date of 8/30. If approved, the expanded indication would allow
Biologics
CBER has released its calendar year 2026 guidance agenda showing it plans to produce 35 draft and final guidances in four categories — Blood and Blood Components (five guidances), Therapeutic Products (19 guidances), Other (two guidances), and Vaccines and Allergenics (nine guidances). In the Vaccines and Allergenics category, CBER says it
Biologics
FDA has issued Pierre Fabre Pharmaceuticals a second Complete Response Letter rejecting its BLA for Atara Biotherapeutics’ investigational cell therapy for a rare and often fatal post-transplant cancer, citing the need for additional clinical evidence. The rejection appears to be one of the first rejections under a new CBER
Biologics
FDA says it is formalizing and more broadly communicating a flexible regulatory approach to chemistry, manufacturing and controls for cell and gene therapies, a move the agency says is intended to speed development of innovative treatments while maintaining product quality standards. An FDA notice says details are being shared on
Biologics
With all the staffing turmoil FDA went through in 2025, drug and biologic approvals dropped below totals in the previous two years. A Fierce Pharma analysis finds that there were 46 novel drug approvals in 2025, compared to 55 in 2023 and 50 in 2024. In addition, there were 18
Biologics
Mainstream media stories at the end of 2025 focused on the ongoing controversy over vaccines brought about by recommendations from CBER director Vinay Prasad and newly-appointed CDER director (the fifth director in 2025) Tracy Beth Hoeg. The Guardian reports that Hoeg’s appointment “could signify a closer partnership between
Biologics
At the 12/3/2025 public meeting on the BsUFA 4 reauthorization, several industry representatives presented their recommendations for matters to be considered in the reauthorization negotiations. CMC regulatory expert Fabiola Gomez (Hyman, Phelps & McNamara) outlines the recommendations in her firm’s FDA Law Blog. The Biosimilar Forum urged
Biologics
FDA has set a PDUFA action date of 10/30 for its review of an Inovio BLA for INO-3107 as a potential treatment for adults with recurrent respiratory papillomatosis (RRP). A company statement says the BLA was filed under the Accelerated Approval pathway. Inovio says the agency acceptance letter
Biologics
FDA has cited a GlaxoSmithKline biologicals manufacturing site in Hungary for significant lapses in deviation investigations and material controls, including failures to assess worst-case conditions for batches later used in the production of widely used vaccines. The just-posted FDA Form 483 followed an inspection conducted from 1/23
Biologics
FDA has issued two separate Form 483 inspection reports citing a range of quality and compliance deficiencies at Catalent Maryland, Inc., highlighting ongoing challenges in the company’s contract biologics and gene therapy manufacturing. The inspections, conducted in April at the company’s Baltimore and Harmans, MD, facilities, identified multiple
Biologics
Sen. Ron Johnson (R-WI) is demanding that FDA turn over records related to an internal review that linked Covid-19 vaccinations to the deaths of at least 10 children, escalating a long-running Republican investigation into vaccine safety and federal oversight during the Biden administration. In a 12/15
Biologics
A 3/31-4/6 FDA inspection at the Microvascular Tissue facility in San Diego, CA, determined that the company’s mVASC, derived from hypodermis recovered from donors and marketed for allogenic use, is an unapproved new drug and an unlicensed biological product. A 12/3 Warning Letter says the
Biologics
FDA has “no plans” to add a Black Box Warning to Covid vaccines, FDA commissioner Marty Makary said in a Bloomberg TV interview, emphasizing that the agency’s focus is on transparency and updated risk assessments rather than broad changes to vaccine policy. Makary said the FDA’s safety and
Biologics
Vanda Pharmaceuticals has filed a BLA for imsidolimab and its use in treating generalized pustular psoriasis, a rare and potentially fatal inflammatory skin disease with no broadly effective long-term therapies. The disorder is driven in many cases by mutations in the IL36RN gene that disrupt the body’s natural
Biologics
FDA is preparing to add a Boxed Warning — the agency’s most stringent safety alert — to Covid-19 vaccines, according to two people familiar with the deliberations. The potential move marks a dramatic escalation in the Trump administration’s ongoing campaign to scrutinize and restrict use of the vaccines, which