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FDA’s rejection of rare disease drugs causes Orphazyme to lay off two-thirds of its staff

Orphazyme, a rare disease biotechnology Layoffs Two-thirds of employees, this is the action taken 10 days after the FDA rejected the company’s main drug candidate.

Orphazyme, headquartered in Copenhagen, has not given up on the drug, arimoclomol. When Orphazyme announced the layoffs on Monday, it said that the company’s restructuring could save cash and enable the company to focus the remaining resources on ensuring that the drug is approved in Europe and evaluating the future prospects of the drug in the United States.

Orphazyme did not specify the details of the layoffs, but before the FDA is expected to approve arimoclomal, the company has been adding staff to prepare and execute the commercial release. According to the company’s annual report, as of the end of last year, Orphazyme employed 141 employees, up from 85 employees at the end of 2019. The number of employees last year included 33 people in pre-commercial and commercial positions. These workers and some workers in other departments are likely to be laid off.

Arimoclomol aims to treat diseases by using “heat shock” proteins, which are produced by the body to prevent cells from becoming toxic due to misfolded proteins, protein aggregation and lysosomal dysfunction. Orphazyme drugs are designed to cross the blood-brain barrier and selectively amplify heat shock proteins. The main disease target is Niemann-Pick type C, which is a lysosomal storage disease that involves the body’s ability to process and recycle fat. This disease can cause tissue damage, including brain tissue. Symptoms include difficulty swallowing, speech and cognitive problems, and loss of motor coordination. There is no cure for this disease and it is usually fatal in the patient’s 20s.

The main goal of a 50-patient Phase 2/3 clinical trial is to assess disease progression based on a scale with five Neimann-Pick disease type C measurements. Improvements in clinical trials are not enough to make clinical trials a success. Orphazyme stated in the regulatory filing that statistical significance was achieved only when three patients with rare genetic mutations that could predict the early onset and rapid progression of Neimann-Pick C type were excluded.

According to Orphazyme, the FDA’s rejection letter requires More data Confirm the company’s interpretation of the results of the Five Domain Assessment Scale, especially the measurement of swallowing. In addition to the single phase 2/3 study, the agency also requested more data to support the drug’s risk-benefit assessment.

On Monday, Orphazyme announced 36 months of data During the Parseghian Scientific Conference on Neimann-Pick Disease Type C Research, the persistence of Almochlorohydrin was shown. These results indicate that patients continue to show benefit based on the same five-domain scale used to evaluate drugs in phase 2/3 testing. A total of 33 patients completed 36 months of treatment. The adverse events in this open-label study are comparable to those observed in the double-blind clinical trial.

The reorganization of the company is not unexpected.In the FDA’s rejection statement, Orphazyme CEO Christophe Bourdon stated that the company will need to “significantly reduce our costs and freeze all company efforts not related to clinical and regulatory activities to support approvals. [Neimann-Pick disease type C]. “

The Committee on Pharmaceutical Products for Human Use is expected to comment on the drug in the fourth quarter of this year. The European Medicines Agency may make a decision in the first quarter of next year.

In addition to layoffs, three Orphazyme board members will also step down. The company stated that Rémi Droller, Martijn Kleijwegt and Anders Hedegaard will resign on June 30 and will not be replaced. The remaining board of directors will have six members. Orphazyme said that its cash position is expected to be 50 million Danish kroner by the end of this year, which is lower than the expected 350 million Danish kroner.

Image: Richard Morrel, Getty Images

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