Grace Therapeutics Inc.’s hopes of becoming the first company in decades to update the standard of care in aneurysmal subarachnoid hemorrhage were delayed after the U.S. FDA issued a complete response letter (CRL) for its GTx-104 NDA, citing manufacturing and nonclinical deficiencies rather than concerns over efficacy or safety.
The U.S. FDA’s recent pledge to make public complete response letters (CRLs) for drugs and biologics that failed to gain approval has encountered some objections from industry regarding the potential for disclosure of trade secrets and proprietary information.
Chiesi Group’s idebenone faced a regulatory setback last month after the U.S. FDA issued a complete response letter (CRL) to the company’s NDA for Leber hereditary optic neuropathy (LHON), a rare inherited disorder that causes sudden vision loss.But a chance missed for Chiesi may be an opportunity for gene therapies, including Gensight Biologics SA’s lenadogene nolparvovec (Lumevoq; GS-010).
