Polyneuron Pharmaceuticals receives EMA orphan drug designation

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20.07.2017

The Biotech Startup Polyneuron Pharmaceuticals AG announces that the European Medicines Agency (EMA) has granted an Orphan Drug Designation for PN-1007 as a novel treatment for anti-MAG Neuropathy. An important milestone in the development of the new therapy.

Anti-MAG neuropathy is a severe and chronically debilitating autoimmune disease of the peripheral nervous system affecting approximately 70’000 patients worldwide. Patients with anti-MAG neuropathy suffer from sensory loss in their arms and legs, neuropathic pain, tremor, and a loss of coordination that can substantially impair mobility. Through progressive damage to the peripheral nerves, which is mediated by anti-MAG autoantibodies, the patients become increasingly disabled in their day-to-day lives until they require full-time care.

To address this problem, Polyneuron developed «PN-1007» as a novel treatment for anti-MAG Neuropathy. The therapeutic glycopolymer PN-1007 is designed as a scavenger for pathogenic anti-MAG autoantibodies, that enables highly efficient antibody neutralization and removal in vivo without adverse immunosuppression (Herrendorff et al. PNAS, 2017). This antigen specific treatment approach offers significant potential not only for an antigen-specific treatment of anti-MAG neuropathy but also for other antibody-mediated autoimmune diseases.

According to the startup, approximately 70’000 people worldwide are suffering from the disorder, yet there is no satisfactory treatment with approval by the EMA. Current therapies are used off-label – that is, without approval by health authorities – and mostly directed to suppress the immune response; they are inefficient and in some cases cause serious side effects.

Thanks to the receipt of the EMA orphan drug designation, the startup will proceed with the development of their novel therapy. “The grant of the designation is a very important milestone for us, says Pascal Hänggi, CSO of Polyneuron.

The EMA orphan drug designation is a status assigned to a medicine intended for use against a rare condition. With the orphan drug designation, EMA offers a range of incentives, including free protocol assistance for SMEs, centralised authorization procedure, and extended market exclusivity.

About Polyneuron
Established as a spinoff from the University of Basel, Polyneuron Pharmaceuticals is developing a ground-breaking drug class for the treatment of autoimmune disorders. Their technology platform “Antibody-Catch” enables the design of drugs that selectively inactivate disease-causing autoantibodies, while leaving the immune system intact. This pioneering therapeutic approach holds great potential for effective and safe treatments for antibody-mediated autoimmune diseases.

Picture: DarkoStojanovic/Pixabay

(RAN)

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