EMA Endorses Alexion’s Strensiq to Treat Rare Bone Disease

The European Medicines Agency recommended under exceptional circumstances Alexion Pharmaceutical’s Strensiq for the long-term treatment of hypophosphatasia, a rare metabolic disorder that weakens bones.

Strensiq (asfotase alfa), the first therapy for the disease, replaces an enzyme that is key to creating and maintaining healthy bones and calcium and phosphate in the body.

The Committee for Medicinal Products for Human Use recommends drugs for marketing authorization under exceptional circumstances when they offer new or improved treatment options and no alternatives exist, but there is not comprehensive safety and efficacy data — as in Strensiq’s case, due to the extreme rarity of the disease.

As a condition of approval, Alexion would have to collect additional data postmarketing and submit it regularly to CHMP for review, the EMA says.

Because Strensiq would be administered to growing children, the EMA is advising a color-coded system for cartons and labels on vials to distinguish various strengths and volumes and ensure adjustments in dosing don’t result in over-dosing or under-dosing.

In a flurry of EMA recommendations for rare disease treatments, Alexion also picked one up for Kanuma (sebelipase alfa), a drug to treat lysosomal acid lipase deficiency, a life-threatening inherited condition that causes growth failure, enlarged liver, diarrhea and poor absorption of nutrients.

Novartis’ Farydak (panobinostat), a multiple myeloma drug that targets enzymes involved in turning genes “on” and “off” within cells also received a nod, as did AbbVie’s Humira for an expanded indication to treat adults with active moderate to severe hidradenitis suppurativa — a severe skin disease — who have failed to respond to conventional systemic treatments. — Kellen Owings