Biologics
CBER recently used a virtual town hall to urge developers of cell and gene therapies to take advantage of early engagements with the agency well before a BLA filing to help avoid delays and common regulatory pitfalls. According to a just posted transcript, officials from CBER's Office of
Biologics
Johnson & Johnson is reporting positive topline results from the three-arm investigational Phase 3 MonumenTAL-6 study evaluating Tecvayli (teclistamab-cqyv) plus Talvey (talquetamab-tgvs), a BCMA and GPRC5D dual antigen targeting regimen, and Talvey plus pomalidomide in adult patients with relapsed or refractory multiple myeloma (RRMM) who received
Biologics
FDA has accepted for priority review a Dyne Therapeutics BLA for zeleciment rostudirsen for treating individuals with Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping. The company says it has requested accelerated approval based on dystrophin as a surrogate endpoint. The agency set a user fee review action target
Biologics
HHS aide Stefanie Spear, a close associate of HHS secretary Robert F. Kennedy Jr. for many years, has been asking FDA to provide access to its Sentinel and BEST surveillance system data to help the HHS staff conduct studies, including into rates of autism and other conditions among vaccinated versus
Biologics
Former CDC chief medical officer Debra Houry said she left the agency after concluding she could no longer defend its scientific integrity, citing widespread political interference in vaccine policy, communications, and data operations, according to a 7/5 CBS News interview aired on Face the Nation. The interview provides a
Biologics
FDA has published a guidance, Recommendations for Assessment of Blood Donor Eligibility, Donor Deferral, and Blood Product Management in Response to an Ebola Disease Outbreak, notifying blood collection establishments of the agency’s determination of Ebola to be a transfusion-transmitted infection. The document includes the agency’s recommendations for
Biologics
CBER has published a Standard Operating Policy and Procedure (SOPP) to guide staff in implementing and maintaining Risk Evaluation and Mitigation Strategies (REMS). The document says the SOPP applies to an initial REMS as part of an original BLA or NDA, or a new REMS needed as part of an
Biologics
FDA has told Capricor Therapeutics that it has scheduled a meeting of the Cellular, Tissue, and Gene Therapies Advisory Committee to review the company's BLA resubmission for deramiocel, an investigational cell therapy for treating Duchenne muscular dystrophy. The panel will meet on 7/29, less than a month
Biologics
The European Commission has opened a formal antitrust investigation into whether Sanofi abused its dominant market position by allegedly conducting a misleading campaign against a rival influenza vaccine marketed by CSL Seqirus. The investigation centers on Sanofi's promotion of its enhanced influenza vaccine, Efluelda, which competes in Europe
Biologics
FDA has accepted for review Replimune's BLA resubmission that is seeking accelerated approval of RP1 (vusolimogene oderparepvec) in combination with nivolumab for treating advanced melanoma, the company announced Friday. The agency classified the filing as a Class 1 resubmission and set a review action target date of 8/
Biologics
A Covid-19 vaccine effectiveness study that was previously blocked from publication in a Centers for Disease Control and Prevention (CDC) journal has now been published in JAMA Network Open, reigniting a debate over both vaccine policy and the methods used to evaluate vaccine performance. The study found that the
Biologics
FDA has given Beam Therapeutics the green light in its IND for BEAM-304, allowing the company to begin clinical testing of its experimental base-editing treatment for phenylketonuria (PKU). The clearance enables the launch of a Phase 1/2 trial evaluating BEAM-304, an in vivo gene-editing therapy
Biologics
The FDA Vaccines and Related Biologic Products Advisory Committee has voted to recommend approval of Moderna’s mFlusiva mRNA flu shot. The Associated Press reports the company is seeking approval for the vaccine’s use in people ages 50-64, along with authorization for use in those aged 65 and
Biologics
Sens. Jeanne Shaheen (D-NH) and Susan Collins (R-ME), co-chairs of the Senate Diabetes Caucus, are calling on FDA to improve access to deceased donor islet cell transplantation for certain individuals with Type 1 diabetes (T1D). In a letter to FDA acting commissioner Kyle Diamantas, the senators say
Biologics
Sen. Ron Johnson (R-WI) has launched a new investigation into federal handling of Covid-19 vaccine safety issues, seeking records from HHS, FDA and CDC related to internal discussions about adding a warning for "myocarditis with fatal outcomes" to mRNA Covid-19 vaccine labeling. In a 6/
Biologics
FDA has reversed course and has told UniQure that data from its ongoing Phase I/II study of AMT-130 could serve as the primary basis for a BLA seeking accelerated approval for the investigational Huntington's disease gene therapy. The regulatory feedback was provided during a recent Type
Biologics
FDA has accepted for review a BLA resubmission from Outlook Therapeutics seeking approval of ONS-5010/Lytenava (bevacizumab-vikg) for treating neovascular age-related macular degeneration, commonly known as wet AMD. The filing focuses on the review of product labeling and represents what the company described as the final step
Biologics
An FDA briefing document released in advance of a 6/18 advisory committee meeting reviewing Moderna's investigational mRNA influenza vaccine, mFlusiva (mRNA-1010), reveals a more favorable outlook than many observers expected after a February Refusal-to-File letter was issued and then reversed shortly thereafter. That application
Biologics
FDA has approved a Lupin BLA for Ranluspec (ranibizumab-hkdz), an interchangeable biosimilar to Genentech's blockbuster eye drug Lucentis. Ranluspec references Lucentis, a vascular endothelial growth factor (VEGF) inhibitor widely used in ophthalmology to treat vision-threatening retinal diseases. The biosimilar is approved for the same major indications
Biologics
Former FDAer Peter Pitts, now president of the Center for Medicine in the Public Interest, writes that ALS (amyotrophic lateral sclerosis) “has become an important stress test for whether American regulatory institutions can adapt to 21st century biomedical science.” Writing in an open-source article in the Journal of the
Biologics
FDA has ordered Takeda Pharmaceuticals to strengthen the safety labeling for its rare disease therapy Adzynma, including the addition of a Boxed Warning, after the agency and the company failed to reach agreement on how to address concerns about potentially life-threatening immune reactions. In a just-posted 5/20
Biologics
FDA has issued a new set of frequently asked questions (FAQ) aimed at helping companies navigate the electronic submission process for products regulated by CBER, offering additional guidance on technical requirements, acknowledgment timelines, and common causes of submission rejections. The FAQ document addresses recurring issues encountered by sponsors submitting biologics