Apellis’s pegcetacoplan – approved for two other indications – will not go forward in amyotrophic lateral sclerosis after a Phase II failure, two days after Wave Life made a similar call in the neurodegenerative disease.

Pointing to data showing benefit in patients who completed 72 weeks of therapy, PTC thinks vatiquinone still could be viable for pediatric FA patients. Restructuring won’t affect Upstaza.

Phase III data for PTC’s sepiapterin shows statistically significant reduction of blood phenylalanine levels in patients with phenylketonuria. BioMarin has the only two approved drugs for PKU.

Only four of 14 voting panelists are regular members of the Cellular, Tissues and Gene Therapies Advisory Committee, with scheduling conflicts precluding participation by many others, the FDA said. Three of the temporary members are veterans of prior muscular dystrophy drug reviews.