Drugs

Dynacure Gets Orphan Status for DYN101

FDA has granted Dynacure an orphan drug designation for DYN101, an investigational antisense medicine designed to modulate the expression of dynamin 2 (DNM2) for treating centronuclear myopathies (CNM). The company expects to initiate a Phase 1/2 study (Unite-CNM) in a few months. The therapy is being developed in collaboration with Ionis Pharmaceuticals, which specializes in RNA-targeted drug discovery.

Centronuclear and myotubular myopathies are serious, rare, life-threatening disorders that affect skeletal muscles from birth, according to Dynacure. “The disease is driven by mutations in multiple genes including MTM1, DNM2 and BIN1 and Dynacure scientists have discovered the link between an increase in DNM2 and the direct cause of the disease,” it says.

Preclinical studies have demonstrated that DYN101 has the potential to be disease modifying in CNM, with what the company calls "compelling" preclinical efficacy in treating animal models of X-linked recessive CNM and autosomal dominant CNM. “Prevention and reversion of the disease was observed with a clear dose-dependent improvement in whole body strength and mice survival,” Dynacure says.

Rasonque OK’d Across KRAS Mutations Despite Clinical Response Differences

FDA granted approval to Revolution Medicines' Rasonque (daraxonrasib) for metastatic pancreatic cancer despite substantial differences in treatment responses among patients with different KRAS mutations and limited evidence of effectiveness in patients without RAS mutations, according to a newly released approval document. The 321-page review shows that FDA considered

FDA Safety Concerns Complicate Plan to Expand Peptide Compounding

The Trump Administration is developing a temporary policy that would allow compounding pharmacies to dispense certain unapproved peptides despite earlier FDA scientific assessments finding insufficient evidence of their safety and effectiveness, setting up a potential conflict between administration policy and the agency's scientific reviewers. Federal officials are considering

DoJ Enforcement Directive Signals More Scrutiny of FDA-Regulated Firms

A new Department of Justice directive identifying violations of the Federal Food, Drug, and Cosmetic Act (FDCA) among potential targets for fraud enforcement could expose FDA-regulated firms to increased federal investigations and criminal prosecutions, according to an analysis by attorneys at Holland & Knight. The 10/1 directive outlines

Guide on Biosimilar Postapproval Manufacturing Changes

FDA has released a final guidance entitled “Postapproval Manufacturing Changes to Biosimilar and Interchangeable Biosimilar Products — Questions and Answers.” The document explains how manufacturers should document and report changes to approved biosimilars, including changes involving manufacturing processes, production facilities, dosage forms and strengths. Regarding information required for postapproval manufacturing changes,

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Rasonque OK’d Across KRAS Mutations Despite Clinical Response Differences

FDA Safety Concerns Complicate Plan to Expand Peptide Compounding

DoJ Enforcement Directive Signals More Scrutiny of FDA-Regulated Firms

Guide on Biosimilar Postapproval Manufacturing Changes