On June 25, 2015, the European Medicines Agency (EMA) recommended granting marketing authorization to Alexion and Novartis for their new rare disease and cancer drugs — a major milestone in delivering these new treatments to patients.
The drugs that were granted these recommendations included 2 new targeted cancer therapies from Novartis: Farydak® (panobinostat), used with other agents for the treatment of relapsed and/or refractory multiple myeloma (an aggressive white blood cell cancer) in adults to delay disease progression; and Odomzo (sonidegib), intended for adults with locally advanced basal cell carcinoma (a common form of skin cancer). Farydak achieved FDA approval in February of this year.
The EMA is fast-tracking evaluation of the orphan drug from Alexion called Kanuma (sebelipase alfa) for the treatment of lysosomal acid lipase deficiency, an inherited, progressive, rare, life-threatening disease. Currently no approved therapy is available for this condition.
Strensiq (asfotase alfa), another orphan drug made by Alexion, has been recommended by the EMA for marketing authorization “under exceptional circumstances”— a special authorization offered for new therapies for diseases that currently have no or limited therapeutic alternatives. In the case of Strensiq, which is intended for the long-term treatment of hypophosphatasia, an inherited, progressive bone disease, the data are limited owing to the extreme rarity of the disease. At present, treatment for patients with hypophosphatasia is confined to calcium supplements and supportive treatment such as pain medication and plaster casts for broken bones.
Both Strensiq and Kanuma are innovative enzyme replacement therapies that would be the first treatments ever available for infants, children, and adults with these disabling and potentially deadly metabolic diseases.
In its next steps, the EMA will determine whether to adopt an EU-wide marketing authorization. Each Member State will then decide upon price and reimbursement based on the potential role of the drug in the context of its national health system. Final decisions from the EMA are expected in the third quarter of 2015.
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