Rare diseases
One of the FDA’s potential approvals this month could break an existing monopoly in the treatment space for a rare growth disorder.
According to reporting from Reuters, reviewers at the agency pointed to an inability to differentiate from placebo to justify rejecting the drug, but an FDA office director approved the drug anyway.
Biohaven is proposing troriluzole for the treatment of spinocerebellar ataxia, a group of rare, genetic diseases that lead to the progressive loss of control over movement.
Harmony Biosciences has paused a mid-stage trial of ZYN002 in 22q11.2 deletion syndrome after the THC-free cannabinoid drug failed to significantly improve social avoidance in a late-stage study in fragile X Syndrome.
Kygevvi is indicated for patients with thymidine kinase 2 deficiency whose symptoms arise by 12 years of age. The disease manifests as muscle weakness and can become life-threatening in severe instances.
Had Pfizer’s Freda Lewis-Hall not stepped in, SpringWorks’ rare disease treatment may never have reached patients. Pharmas can act now to help find the next Gomekli.
Vinay Prasad, chief of the FDA’s Center for Biologics Evaluation and Research, is planning to publish a paper this month to outline his office’s thinking on accelerating gene editing reviews.
Already sky high after the success of its limb-girdle muscular dystrophy therapy, reported on Monday, BridgeBio’s shares rose nearly 8% more as encaleret balanced calcium levels in patients with a genetic thyroid disorder.
BioMarin no longer expects to hit its $4 billion revenue target by 2027, citing various market factors such as impending competition for achondroplasia therapy Voxzogo and the divestment of the gene therapy Roctavian.
Intellia earlier this year reported a similar grade 4 liver enzyme elevation associated with the gene therapy nexiguran ziclumeran, though analysts at BMO Capital Markets at the time brushed it off as a “non-concern.”
PRESS RELEASES