Orphazyme Preps to File European Marketing Application for Experimental Niemann-Pick disease Type C Drug

According to a press release from Danish biopharmaceutical company Orphazyme, the Company intents to proceed with a marketing authorisation application (MAA) for its experimental Niemann-Pick disease type C treatment arimoclomol. If marketing authorization is approved, arimoclomol will be available to treat Niemann-Pick type C patients in every European Union member state.

Following a consultation with the European Medicines Agency (EMA), Orphazyme made plans to file an MAA in the first half of 2020. This summer, Orphazyme will meet with officials from the United States’ Food and Drug Administration to discuss a potential American rollout.

About Niemann-Pick Type C

Niemann-Pick type C (NPC) is a rare genetic disorder characterized by the body’s inability to effectively transport fatty substances (like cholesterol and other lipids) in cells. These fats accumulate in vitally important tissues around the body, including the brain, nerves, bone marrow, and sometimes lungs.

The results are often devastating. Older studies suggest that most individuals with Niemann-Pick C don’t live past 25 years of age, though this figure may not be accurate due to a recent increase in late-onset diagnoses.

Currently, treatment focuses on alleviating symptoms — but there is no cure.

Marketing Authorization Application for Arimoclomol Likely in 2020

The conclusion of a 12-month open-label extension study of arimoclomol’s efficacy in NPC patients is scheduled for some time in the second half of this year. This study must be included as a component of the full marketing authorization application.

In a phase II/III clinical study of the drug, data collected suggested that patients administered arimoclomol in combination with the standard of treatment for Niemann-Pick Type C enjoyed a 74% overall reduction in disease progression. Additionally, study participants in the arimoclomol arm were 16% less likely to experience a serious progression in the severity of their condition (10.7% versus 26.7% of the arimoclomol group).

After the MAA goes through, the Committee for Medicinal Products for Human Use (CHMP) — the EMA’s human medicine authority — will take up to seven months to scientifically assess and evaluate the proposed treatment and all relevant data. After that, the European Commission (the governing administration of the European Union) takes up to an additional two months to form their decision based on the CHMP’s scientific evaluation of the study.

That means despite the promising headline, arimoclomol may not be marketed in Europe until 2021. That is, of course, if it’s approved at all. Orphazyme representatives plan to meet with FDA officials this summer to discuss a potential pathway to American approval.


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