International Niemann–Pick Disease Alliance

Positive Review of Paediatric Investigation Plan (PIP) for AIDNPC

NEWS RELEASE – Copenhagen, 10 February 2016

Positive Review of Paediatric Investigation Plan (PIP) for AIDNPC . EMA’s Paediatric Committee (PDCO) recommends that the Committee for Medicinal Products for Human Use (CHMP) approve Orphazyme’s PIP for use of arimoclomol in treating patients suffering from Niemann-Pick disease type C (NP-C).

The PIP is an overall development plan that is reviewed by the Paediatric Committee (PDCO) of the European Medicines Agency. A PIP ensures that the intervention clinical study with arimoclomol in NP-C will generate the data in compliance with PDCO to support a marketing authorisation of the medicine for children in Europe.

“This favourable opinion on the AIDNPC programme by the Paediatric Committee is an important milestone for Orphazyme and the patient community toward making a new efficacious treatment available for Niemann-Pick disease type C” says Anders M. Hinsby, Chief Executive Officer of Orphazyme.

As part of its AIDNPC clinical programme in NP-C, Orphazyme has initiated an observational study involving patients from Europe and recruitment is well under way.

For further information:
Anders M. Hinsby, CEO in Orphazyme ApS

About Orphazyme ApS

Orphazyme ApS is a Danish biopharmaceutical company, which develops paradigm-changing medicines for the treatment of genetic diseases. The lead program is entering clinical development as treatment for lysosomal storage disease. This family of genetic disorders includes NP-C and consists of more than 45 diseases, often affecting children, most of which are currently untreatable and often fatal. Orphazyme has raised more than €37m in seed, Series A and Series B financing from a strong investor base, which includes Novo A/S, Sunstone Capital, Aescap Venture, Kurma Partner and IDInvest. For more information, please visit

Find out more about Orphazyme at

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