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First von Willebrand disease therapy heads new FDA approvals

Baxalta's Vonvendi set for an early 2016 launch in the US

FDA headquarters 

The traditional flurry of FDA approvals at the end of the year seems already to be underway, with a green light for three new drugs yesterday.

Heading the list is Baxalta's Vonvendi (recombinant von Willebrand factor; BAX 811) - a glycoprotein found in the blood, which plays a key role in clotting - for the bleeding disorder von Willebrand disease (VWD).

VWD is the most common inherited bleeding disorder and affects around 1% of the US population, according to the FDA. Symptoms include severe bleeding from the nose, gums, and intestines, as well as into muscles and joints.

In trials involving 69 VWD patients, Vonvendi was shown to be safe and effective for the on-demand treatment and control of bleeding episodes from a variety of different sites in the body, with one infusion typically enough to control more than 80% of bleeds. 

Baxalta said in a statement that it plans to launch Vonvendi in the US next year, with a filing in Europe and other markets in 2017. It has not yet divulged its pricing plans for the product.

The approval is Baxalta's second for a brand new product since separating from sister company Baxter International, and comes as the company is facing a takeover attempt by Irish pharma company Shire.

Last month it secured FDA approval for long-acting haemophilia A therapy Adynovate (formerly BAX 855), a follow-up to its big-selling Advate brand. Both new drugs are considered to be critical assets for the company as it faces increased competition in the bleeding disorder market from Biogen.

Staying in the rare disease area, the FDA has also given a green light to Alexion's Kanuma (sebelipase alfa), a treatment for patients with lysosomal acid lipase (LAL) deficiency, after a delay of three months to its review.

Kanuma is the first approved treatment in the US for the enzyme deficiency disease, which causes a build-up of fats in cells that can lead in turn to liver and cardiovascular complications. 

An early-onset form of the disease typically leads to death in the first year of life. Patients with a milder, later-onset form of LAL deficiency can survive for longer but remain at high risk of organ damage and premature death.

In trials, giving the treatment to infants with LAL deficiency allowed 67% of patients to survive beyond 12 months, while children and adults had meaningful improvements in multiple disease-related liver and lipid abnormalities.

The drug is manufactured in the egg whites of genetically -modified chickens, and was acquired by Alexion along with Synageva in an $8.4bn deal announced in May. It has also been approved in the EU and is filed in Japan.

Finally, Teva and Eagle Pharmaceuticals have been given the green light from the FDA for Bendeka (bendamustine), a new formulation of the drug that can be administered via a shorter, lower-volume infusion - for chronic lymphocytic leukaemia and B-cell non-Hodgkin's lymphoma.

Teva has US commercialisation rights to Bendeka under a deal with Eagle signed in February, and says it plans to launch the therapy during the first quarter of 2016.

Article by
Phil Taylor

9th December 2015

From: Regulatory



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