Recent Advances in Medical Treatments and Regulatory Approvals
Monoclonal Antibody Therapy for Early Alzheimer’s Disease
On November 16th 2024, the CHMP granted Eisai Europe and Biogen a positive opinion for their monoclonal antibody therapy aimed at treating early Alzheimer’s disease.
Breakthrough in Bladder Cancer Treatment
Balversa (erdafitinib) has been highlighted as the first and only bladder cancer therapy targeting FGFR3 alterations, showing an increase in overall survival from 7.8 months to 12.1 months as of November 8th 2024.
FDA Updates on Rare Disease Innovation
CDER Director Patrizia Cavazzoni and CBER Director Peter Marks provided an update on the rare disease innovation hub in a new FDA Voices blog post on November 1st 2024.
New Treatment for ATTR
A treatment for ATTR in certain adults with polyneuropathy was recommended for approval across the European Union and had gained regulatory approval in the United States at the end of 2023, as reported on October 31st 2024.
Pfizer’s RSV Vaccine Approval
Pfizer’s bivalent RSV prefusion F vaccine was approved to prevent lower respiratory tract disease in adults 18 to 59 years of age at increased risk on October 23rd 2024.
Advances in Hemophilia A Treatment
As many as 30% of people with severe hemophilia A develop inhibitors such as TFPI, which can limit the effectiveness of replacement therapy, a concern highlighted on October 22nd 2024.
CHMP Recommendations
The agency’s CHMP recommended drugs for hemophilia, two biosimilars for psoriasis, and two flu vaccines at their October meeting on October 18th 2024.
Neurological Treatments Approved
Two drugs, Miplyffa (arimoclomol) and Aqneursa (levacetylleucine), were approved by the FDA to treat neurological symptoms associated with Niemann-Pick disease, type C in adults and children on September 25th 2024.
New Biologic for Atopic Dermatitis
The FDA approved EBGLYSS (lebrikizumab-lbkz), a new biologic treatment for moderate-to-severe atopic dermatitis, on September 24th 2024.
Phase III Trial Results for Imfinzi
Phase III trial results showed that Imfinzi (durvalumab) in combination with chemotherapy reduced the risk of recurrence, progression, or death by 32% in patients with resectable non-small cell lung cancer before and after surgery on August 29th 2024.
NexoBrid Authorization
NexoBrid, a topically administered biological product, is now authorized for use in all age groups in the United States, European Union, and Japan as of August 19th 2024.
High Response Rate for Breyanzi
Breyanzi demonstrated an overall high response rate in adults with relapsed or refractory follicular lymphoma in a Phase II study on August 19th 2024.
Yorvipath for Continuous PTH Exposure
Yorvipath, a prodrug of parathyroid hormone (PTH) (1-34), designed for once-daily administration, was highlighted on August 14th 2024.
Enhertu’s Global Approvals
Enhertu, an antibody-drug conjugate developed by AstraZeneca and Daiichi Sankyo, has been approved for various groups in more than 65 countries since their agreement in March 2019, as reported on August 14th 2024.
Epinephrine Nasal Spray Approval
An epinephrine nasal spray was approved for the emergency treatment of type 1 allergic reactions in adults and children on August 13th 2024.
STADA and Alvotech Launch Uzpruvo
STADA and Alvotech launched Uzpruvo in Europe, the first approved biosimilar to Stelara (ustekinumab) in the European market on July 30th 2024.
GSK’s MAA for Blenrep
GSK's MAA is supported by an interim analysis of Phase III trials, demonstrating significant progression-free survival benefit and positive overall survival trends from Blenrep combination therapies on July 25th 2024.