Regulatory Filings and Submissions

FDA has accepted Sanofi’s sBLA for the mAb and granted it priority review status for the targeted treatment of bullous pemphigoid.

The approval provides greater access to insulin treatment options.

The European Commission has approved Biocon Biologics’ YESINTEK, a biosimilar referencing Janssen Biotech’s Stelara (ustekinumab).

This expanded label allows IZERVAY (avacincaptad pegol intravitreal solution) to be used without a limitation on duration of dosing for treating geographic atrophy.

The approval of an ADCETRIS combination regimen is based on positive data from a Phase III trial where the combination regimen demonstrated a clinically meaningful reduction in the risk of death.

The company plans to assess alternate payloads for its gene therapy program, which will shift its timeline.

On the back of FDA’s designation, March Biosciences is preparing to advance MB-105, a CD5-targeted CAR-T cell therapy, to a Phase II clinical trial in early 2025.

The approval makes Susvimo the first and only continuous delivery treatment for diabetic macular edema.

The committee’s recommendation was based on results from a Phase III trial that demonstrated a 27% reduction in the risk of death versus placebo.

The UK MHRA has granted approval for garadacimab for use in preventing angioedema attacks in patients 12 years old and older who have hereditary angioedema.

The approval makes Enhertu (fam-trastuzumab deruxtecan-nxki) the first HER2-directed therapy in the United States for treating HER2-low or HER2-ultralow metastatic breast cancer.

FDA has approved an additional indication for LEQEMBI (lecanemab-irmb) as a once-every-four-weeks maintenance therapy for early Alzheimer’s disease.

The agency’s newly issued letter to Sanofi amends one sent in December 2024, and stems from inspections at its Massachusetts drug manufacturing facility in June and July 2024.

CNP-104 is a biodegradable nanoparticle that previously received fast track designation from FDA in January 2022, which would eventually make it eligible for accelerated approval and priority review.

This latest fast track designation marks the second such designation by FDA for J&J’s tau-directed investigational therapies for treating Alzheimer’s disease.

The approval makes HYQVIA [Immune Globulin Infusion 10% (human) with Recombinant Human Hyaluronidase] the first and only facilitated subcutaneous immunoglobulin to be approved in Japan to treat these disorders.

The approval of Hikma Pharmaceuticals’ liraglutide injection marks the first generic version of Victoza, a GLP-1 receptor agonist.

EMA and HMA have published revised guidelines on identification of commercially confidential information and personal data used in marketing authorization applications.

EMA's committee gave recommendations for marketing authorization of 17 drugs in December, which include therapies for rare heart conditions, anemia, and liver disease.

The agency will review the company’s supplemental new drug application for vutrisiran, a treatment for ATTR amyloidosis with cardiomyopathy.

The approval of eladocagene exuparvovec-tneq (Kebilidi) marks the first FDA approval for a gene therapy to treat AADC deficiency.

Eisai Europe and Biogen have received a positive opinion for the monoclonal antibody therapy to be used as a treatment of adult patients with early Alzheimer’s disease.

Pfizer’s bivalent RSV prefusion F vaccine has been approved by FDA to prevent lower respiratory tract disease resulting from the respiratory syncytial virus in adults 18 to 59 years of age who are at increased risk.

As many as 30% of people living with severe hemophilia A develop inhibitors such as TFPI, which can be produced by the body’s immune response to clotting factors in replacement therapy, often limiting the effectiveness of that therapy.

The agency’s CHMP recommended drugs to treatments for hemophilia, two biosimilars for psoriasis, and two flu vaccines at their October meeting.

The draft joint network strategy for the EU until 2028 will be open for public consultation until Nov. 30, 2024.

The agency is working with the European medicines regulatory network to improve the assessment and approval process for new medications.

FDA’s Darby Kozak provided commentary on the anniversary of the Drug Price Competition and Patent Term Restoration Act of 1984.

Approvals were recommended for treatments of ovarian cancer, lung cancer, bleeding disorders, respiratory disease, and more.

By approving EBGLYSS (lebrikizumab-lbkz), FDA has made available a new biologic treatment for moderate-to-severe atopic dermatitis that is not well controlled with topical treatments.