Novartis snaps up Priority Review Voucher for $130m

Novartis has got its wallet out to pick up a Priority Review Voucher (PRV) from Ultragenyx, forking out $130 million as part of the trade.

The voucher will allow Novartis to speed up the review by the FDA of a pipeline drug of its choosing. In return, Ultragenyx receive a lump sum of $130 million for its development of Mepsevii – an enzyme replacement therapy for patients with mucopolysaccharidosis VII, a lysosomal storage disorder (LSD).

The condition afflicts only a small number of newborn children, estimated at 1 in 250,000. Since the treatments approval, however, its sales have not been particularly strong and the biotech reported a net loss of $79.2 million.

The $130 million, though not as high as AbbVie paid for United Therapeutics Corp’s voucher ($350 million), is still a significant sum that will ease the biotech’s financial situation as it waits on the FDA’s decision on its lead candidate, burosumab.

Burosumab is a prospective treatment for X-linked hypophosphatemia, a condition where serum phosphate levels in the blood are too low – often leading to rickets.

Phase 3 data on the drug appears strong, as it substantially outperformed placebo in increasing levels of serum phosphorous and improved clinical symptoms of the condition; as a result, the treatment is likely to gain approval early next year and is touted as a potential blockbuster treatment.

"The sale of the PRV provides us with an important source of non-dilutive capital to help advance our pipeline of rare and ultra-rare therapies, and accelerates the availability of these potential therapies to patients," said Emil D. Kakkis, Chief Executive Officer and President of Ultragenyx.

For Novartis, the deal means it can speed a drug candidate through the regulatory process, by having it assessed within six months – shaving four months off the normal procedure. The major question is which treatment does Novartis have in mind to rush through the process?