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BioMarin's Brineura Approval Shows FDA's Open Door For Orphan Drugs

 
• By Emily Hayes and Sue Sutter

Filing supporting Brineura was supported by a tiny, single-arm study and had no advisory committee review, the latest sign of the US regulatory agency's willingness to speed orphan drugs for serious pediatric diseases.

Rapid development and FDA approval of BioMarin Pharmaceutical Inc.'s Brineura (cerliponase alfa) for a rare, life-threatening form of Batten disease afflicting children shows that despite some past controversy, the regulatory doors are still wide open for orphan drugs.

The company announced April 27 that the US FDA approved the drug for symptomatic pediatric patients who are at least three years of age and have late infantile neuronal ceroid lipofuscinosis type 2 (CLN2), a type of Batten disease that is also known as tripeptidyl peptidase 1 (TPP1) deficiency

BioMarin's filing was supported by a single-arm study of 22 evaluable patients, with efficacy compared to a natural history cohort

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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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US FDA Vaccine Framework Coming In Weeks, But Details Vague

 
• By Sarah Karlin-Smith

New CBER Director Prasad will "unleash a massive" framework on vaccines, FDA Commissioner Marty Makary said 15 May, raising questions about the impact on products nearing approval.

Managed Access Agreement Fails To Help BioMarin’s Brineura Secure Routine Reimbursement In England

 
• By Francesca Bruce

BioMarin’s ultra-rare disease drug Brineura has breached cost-effectiveness thresholds for highly specialized treatments in England and looks set to be denied routine funding on the National Health Service.

Inclusive By Design: UK Pilots Diversity Plans For Drug & Device Trials

 
• By Vibha Sharma

The UK has issued clearer guidance to help drug and medical device sponsors demonstrate how they intend to include a diverse and relevant range of participants in their clinical trials.