Rare diseases
The regulator has received reports that a group of patients treated with Adzynma had neutralizing antibodies against the protein the therapy replaces.
Korro Bio is moving back to square one as a preclinical biotech after the failure of KRRO-110 in alpha-1 antitrypsin deficiency. The company’s stock is down 80% on all the news.
The plausible mechanism pathway “could accelerate gene therapy/editing development,” analysts at William Blair said Thursday, while adding that additional clarity is needed.
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.
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