Novartis Buys Rare Drug Voucher for $130M From Ultragenyx

Novartis picked up a priority review voucher in a $130 million deal with California-based Ultragenyx Pharmaceutical Inc.The PRV is for rare pediatric diseases.

Ultragenyx earned the voucher in November when the U.S. Food and Drug Administrationapproved MEPSEVII (vestronidase alfa) for the treatment of children and adults with Mucopolysaccharidosis VII (MPS VII, Sly syndrome). Ultragenyx Chief Executive Officer Emil Kakkis said the sale of the PRV will provide the company with an "important source of non-dilutive capital" that will be used to advance the company’s pipeline of rare and ultra-rare therapies.

Earlier this month, Ultragenyx announced positive results from its Phase III study of burosumab in adults with X-linked hypophosphatemia (XLH). Patients treated with burosumab for 48 weeks showed sustained maintenance of normal serum phosphorus levels and further improvement in stiffness, physical function and pain, Phase III data showed. The positive Phase III data keeps Ultragenyx on track for the U.S. Food and Drug Administration to approve burosumab for XLH. The FDA set a PDUFA date for April 17, 2018.

The vouchers can speed up the time the FDA reviews a drug by up to four months. That reduces the amount of time for a regulatory review to about six months from the time a New Drug Application is filed. That shortened timeline can be crucial when companies are competing to be first to market with a new therapy.

For Novartis, this is the second PRV it has acquired in the past few months. In September, the FDA awarded the company with the PRV following approval of Kymriah, the company’s CAR-T therapy for the treatment of B-cell acute lymphoblastic leukemia. But what the company plans to do with either of the vouchers remains a mystery. A Novartis spokesperson told Endpoints News that any speculation on what the company intends would be "premature."

In November, BioSpace highlighted four Novartis treatments that could be potential blockbusters for the Swiss company. Among the treatments is RTH258 (brolucizumab), an ophthalmology drug. During a presentation at the American Academy of Ophthalmology, RTH258 showed superiority versus Regeneron's aflibercept (Eylea) in key secondary endpoints reflective of disease activity in patients with nAMD. Patients treated with RTH258 showed fewer signs of specific disease activity than patients treated with aflibercept. Novartis said it expects to file for regulatory approval for RTH258 in nAMD by the end of the fourth quarter of 2018.

For now though, Novartis will remain mum on what it intends to do.