Quality Systems

Articles

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.

FDA Rare Disease Innovation Moves Forward

Patrick Lavery is an Editor for the MJH Life Sciences brands 

. Previously at MJH, he filled the same role for 

The treatment for ATTR in certain adults with polyneuropathy had just been recommended for approval across the European Union and had gained regulatory approval in the United States at the end of 2023.

AstraZeneca’s Wainzua Gains Another Approval in England and Wales

Vyalev, also known as Produodopa, has been approved in 35 countries, with more than 4200 patients having started treatment.

FDA Approves AbbVie Therapy for Adults with Advanced Parkinson’s Disease

Pfizer’s bivalent RSV prefusion F vaccine has been approved 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.


FDA Approves RSV Vaccine

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.

CHMP Gives Positive Opinion to Novo Nordisk Hemophilia Treatment

Wainzua had been previously approved for use by patients with ATTRv-PN in the United States, under the brand name Wainua.

AstraZeneca’s Polyneuropathy Treatment Recommended for EU Approval

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

Several Medicines Recommended by EMA’s CHMP in October

The agency has reconfirmed its recommendation to not give positive opinion to Translarna (ataluren) after a reexamination of the available data.

EMA Denies Authorization of Duchenne Muscular Dystrophy Treatment, Again

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

EMA and HMA Publish Network Strategy for Public Consultation

Cheryl Barton is director of PharmaVision, 

Regulators, EMA and HMA, have published principles and recommendations 
on the use of LLMs, which are being increasingly used for daily tasks.


Regulatory Oversight and Compliance