Orphazyma A/S Granted Rare Pediatric Disease Designation for NPC Treatment


Arimoclomol, in development by Danish biotech Orphazyme A/S for the treatment of Niemann-Pick Disease Type C, was granted rare pediatric disease designation by the U.S. FDA this morning.

Arimoclomol, in development by Danish biotech Orphazyme A/S for the treatment of Niemann-Pick Disease Type C (NPC), was granted rare pediatric disease designation by the U.S. Food and Drug Administration (FDA) this morning, the company announced.

Arimoclomol, an orally available small molecule, is currently being investigated in a clinical Phase II/III trial as a potential treatment for the rare disease.

The NPC gene codes for a protein that is used that is used to transport cholesterol and other lipids between cells. The genetic lysosomal storage disease is classified by the buildup of cholesterol and other lipids between cells. While the time of onset is variable, NPC is most commonly diagnosed in childhood and adolescence.

In patients, neurological symptoms like seizures, delayed motor development, and cognitive problems frequently progress from a buildup of cholesterol in the brain. This gradual neurological pathology is the trademark of NPC and is often results in disability and premature death.

“We are very pleased with the FDA’s continued support for the development of therapies for children with rare diseases,” said Anders Hinsby, CEO of Orphazyme in a press release. “Arimoclomol has now been granted orphan drug, fast track, and rare pediatric disease designations by the FDA, which underpin the high unmet need in NPC, providing acknowledgement of the arimoclomol program. We look forward to continue working closely with the FDA in our effort to provide a treatment option for patients suffering from NPC.”

The drug has exhibited a favorable safety and tolerability record in humans, and 7 different phase 1 clinical studies have been conducted in healthy volunteers. It is recommended that arimoclomol is administered orally, 3 times daily. It can be easily dissolved in liquids or food for best possible patient comfort and compliance.

The rare pediatric disease designation allows for arimoclomol to receive certain incentives as it moves through the pipeline. If a drug candidate with this designation receives marketing approval in the U.S. and meets certain criteria, the FDA will issue a Priority Review Voucher to sponsor the company.

Orphazyme has previously received orphan drug designation for arimoclomol in NPC, amyotrophic lateral sclerosis (ALS), and sIBM from both the FDA and the European Medicines Agency (EMA).

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