FDA wants another trial in ultra-orphan Barth syndrome, but could not identify a feasible design – so Stealth submitted the NDA anyways, based on a retrospective natural history control trial
Stealth BioTherapeutics Inc. is gambling that Barth syndrome patients and advocates can help carry its NDA for elamipretide forward despite the US FDA’s recommendation that a second clinical trial be conducted before submission.
“Although the FDA has recommended that additional controlled data be generated to support NDA review, neither the FDA nor the...
The first “Rare disease Innovation, Science and Exploration (RISE) meeting to discuss the choice of control arms for studies in small populations will be Sept. 3 after plans were delayed by ongoing changes at the FDA.
The Pink Sheet takes an exclusive look at the complete response letter, which shows the information on application deficiencies that could become widely available if FDA leaders succeed in releasing CRLs in real time.
The Pink Sheet viewed Stealth's complete response letter and other documents indicating the FDA appears willing to grant accelerated approval to elamipretide before a confirmatory trial begins enrolling. But the company says it needs a two-month review of the NDA resubmission to survive.
Chiesi’s orphan drug Raxone has secured an English funding recommendation for the treatment of Leber's hereditary optic neuropathy, a decade after the orphan drug was approved for marketing. Chiesi told the Pink Sheet about its “long, and often challenging” road to reimbursement.
