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Chiesi's ultra-rare genetic disorder therapy wins European backing

Lamzede is set to become the first approved therapy for Alpha-Mannosidosis

ChiesiChiesi Group’s Lamzede (velmanase alfa) has been recommended for approval in Europe, putting it on course to become the first pharmacological therapy to reach the market for Alpha-Mannosidosis (AM).

The positive opinion from the Committee for Medicinal Products for Human Use (CHMP) was issued under ‘exceptional circumstances’. This aims to grant access for extremely rare disorder treatment when traditional large-scale clinical studies are not feasible.

In Lamzede’s case it has been investigated in 33 patients, both adults and children, with AM, an ultra-rare, genetic lysosomal storage disorder caused by the absence or malfunction of the alpha-mannosidase enzyme.

Patients with this disorder can experience symptoms such as facial coarsening, intellectual disability, progressive motor function disturbances, hearing impairment and impaired speech.

Lamzede, which was acquired when the Italian group bought Zymenex in 2013, is administered weekly as intravenous infusions, and the medicine works by replacing the missing or malfunctioning enzyme.

It’s the second most advanced product from Chiesi’s rare disease unit after its LSCD therapy Holoclar claimed Europe’s first stem cell therapy approval.

Paolo Chiesi, vice president and head of R&D for Chiesi Group, said: “The positive opinion is an important milestone for brining this therapeutic option to patients affected by AM Across Europe.”

Lamzede, which has been investigated in 33 patients, both adults and children, and the Italian group expects a licencing decision by the European Commission in the second quarter of 2018.

Article by
Gemma Jones

31st January 2018

From: Regulatory

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