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Sanofi’s rare blood disorder drug Cablivi set for European approval

Rare disease drug among notable CHMP recommendations

sanofi genzyme

Sanofi is closing in on EU approval of Cablivi for acquired thrombotic thrombocytopenic purpura (aTTP) – the lead asset behind its €9.3bn acquisition of Belgium’s Ablynx earlier this year.

The EMA’s Committee for Medicinal Products for Human Use (CHMP) gave its blessing to Cablivi (caplacizumab) at its monthly meeting last week, along with three other rare disease therapies, two CAR-T therapies and the usual clutch of new drugs and line extensions.

Cablivi is the first therapeutic to be recommended for approval in Europe for aTTP, a rare autoimmune blood disorder that causes extensive clot formation in small blood vessels around the body, starving organs of oxygen. At the moment, the only treatment for aTTP patients is plasma exchange – which is both expensive and risky – and potent immunosuppressive drugs that have serious side effects.

Europe looks set to be the first market for the new drug, ahead of the US where it has not yet been submitted for approval, according to Sanofi. Analysts at Jefferies think Cablivi could be a game-changer in aTTP treatment and generate sales of up to $500m a year.

Among the other recommendations, Ultragenyx picked up a positive CHMP opinion for its mucopolysaccharidosis type VII drug – Mepsevii (vestronidase alfa) – which was approved in the US last November.  The drug acts as an enzyme replacement therapy for the ultra-rare lysosomal storage disorder, which causes the accumulation of complex carbohydrates in tissues and widespread organ damage.

Shire unit Baxalta got a green light for Veyvondi (vonicog alfa) for the treatment of von Willebrand disease, the most common inherited bleeding disorder. The drug should be used as when standard therapy desmopressin is ineffective or contra-indicated, according to the CHMP. Vonicog alfa was approved in the US as Vonvendi in 2015

Jazz Pharmaceuticals’ Vyxeos (liposomal daunorubicin/cytarabine) was the fourth orphan disease drug to be recommended by the CHMP, getting the nod for the treatment of newly-diagnosed acute myeloid leukaemia (AML).

If fully approved by the European Commission – which almost invariably follows CHMP backing – the drug will become the first chemotherapy option specifically for therapy-related AML or AML with myelodysplasia-related changes in the EU, according to Jazz, which got FDA approval for the drug last August.

In addition to the orphan drug approvals, the CHMP also gave positive opinions to Gruenenthal’s gout treatment Duzallo (lesinurad/allopurinol), a follow-up to the company’s Zurampic (lesinurad) product. European rights to both drugs were acquired from AstraZeneca in a $230m deal two years ago.

There was good news too for Puma Biotech after the committee backed approval for its adjuvant HER2-positive breast cancer drug Nerlynx (neratinib), having rejected it at an earlier meeting in February. Nerlynx was approved by the FDA in July 2017.

Article by
Phil Taylor

2nd July 2018

From: Regulatory



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