FDA rejects Orphazyme rare disease drug

June 18, 2021

Denmark-based Orphazyme said its application for FDA approval for arimoclomol as a treatment option for Niemann-Pick disease type C (NPC) was turned down with a complete response letter.

Orphazyme's lead candidate, arimoclomol is a heat shock protein amplifier intended for the treatment of NPC, a rare, relentlessly progressive, neurodegenerative disease. There are currently no approved treatments for NPC in the U.S.

According to the CRL, the FDA needs additional qualitative and quantitative evidence to show the drug's effectiveness. The agency has asked for additional data to strengthen evidence beyond the single phase 2/3 clinical trial to support the benefit-risk assessment of the NDA.

The drugmaker said it would remain in dialogue with the FDA and will also continue to seek approval for the treatment in Europe.

Orphazyme is yet to have a drug approved, but its stock made the rounds in social media last week, at one point surging from $5 to $77 per share amid Reddit hype. Shares have since fallen back down to $8 after news of the FDA rejection.

Read the press release