PTC Therapeutics Sells Priority Review Voucher for $150M Following Phase 2 ALS Trial Failure

Phase 2 ALS Trial Failure:
PTC Therapeutics reported that its Phase 2 trial for an ALS treatment failed to meet its objectives14.

Sale of Priority Review Voucher:
The company sold a priority review voucher, which it had obtained from the FDA approval of Kebilidi, for $150 million45.

Monetization Strategy:
PTC had planned to monetize the voucher, and the sale aligns with this strategy5.

Context:
The sale of the priority review voucher comes at a time when the FDA's planned sunsetting of the rare pediatric disease voucher program has led to increased interest in such vouchers2.

Impact:
The failure of the ALS trial and the sale of the voucher reflect the challenges and strategic decisions biotech companies face in drug development and regulatory processes.

Sources:

1. https://endpts.com/applied-therapeutics-rare-disease-drug-gets-a-crl/

2. https://www.biopharmadive.com/topic/fda/

4. https://www.fiercebiotech.com/biotech

5. https://www.fiercebiotech.com/biotech/ptc-sells-priority-review-voucher-kebilidi-approval-150m