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‘Warp Speed’ For Rare Diseases: Gene Therapy Pilot Also Includes Neurodegenerative Drugs

 
• By Grace Moser

A plan developed by the US FDA biologics center to test ‘Operation Warp Speed’ style communication to support development of gene therapies is launching – and the scope has broadened to include drugs targeting ALS and other neurodegenerative diseases.

Warp Speed
• Source: Shutterstock

The US Food & Drug Administration will be offering “Operation Warp Speed” style interactions for up to six IND sponsors who are pursuing cell and gene therapies for rare pediatric disease or drugs for rare neurological disorders.

FDA announced the opportunity to participate its new Support for clinical Trials Advancing Rare disease Treatment, or START, pilot program at the end of September. The pilot will test “more rapid, ad hoc communication” between FDA and the sponsors, modeled on the “Warp Speed” approach taken to COVID product development during the pandemic

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US FDA Rare Disease Case Studies Provide Development Models For Sponsors

 
• By Laura Helbling

Sanofi’s Xenpozyme and Sentynyl’s Nulibry are the first two case studies the FDA is using to continue educating rare disease sponsors on best practices.

House Budget Bill Includes Delayed Orphan Fix, Also Risks Downstream Cell and Gene Coverage

 
• By Sarah Karlin-Smith

The first two classes of negotiated drugs under the Inflation Reduction Act would not benefit from a rare disease adjustment House Republicans included in their reconciliation package.

Tougher Approval Standards May Follow Vinay Prasad’s Appointment To Lead US FDA’s CBER

 
• By Sarah Karlin-Smith

Industry is concerned that Prasad may make regulatory flexibility tougher to obtain for cell and gene therapy, while vaccine and public health advocates are angry about Prasad’s criticisms of US COVID-19 policies.

Sponsors Like START Rare Disease Pilot, Will Prasad Maintain Its Momentum?

 
• By Kate Rawson

Sponsors reported faster development times for products that joined the pilot program intended to speed rare disease treatments in CBER.

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