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Progress on Drug for Orphan Disease


Orphazyme, a Danish biotech company working on the development of orphan drugs, recently completed a new series B capital round of 20 million Euro. Kurma Partners and Idinvest Partners joined the existing investor syndicate of Novo A/S, Sunstone Capital and Aescap Venture.

 The company is working on Arimoclomol, the leading drug candidate for the treatment of the genetic lysosomal storage disease Niemann-Pick type C (NP-C). The disease just received orphan drug status from the FDA. Orphazyme plans to initiate a phase 2/3 clinical trial with the drug by the end of second quarter of this year.

About 500 cases of  NP-C  have been diagnosed worldwide and misdiagnoses could mean there are several more. The majority of children with NPC die before age 20 and many die before the age of 10. Later onset of symptoms can lead to longer life spans but it is extremely rare for any person with NPC to reach age 40. Once a medication is developed, patients would have to remain on it throughout their lives.

In 2014, Orphazyme was named Ernst & Young’s Entrepreneur of the Year.

Source: BioPeople