Novartis buys Pharming priority review voucher for a song

Pharming has agreed a deal to sell a recently awarded priority review voucher (PRV) to Novartis for around $21 million - well below the market rate.

The price of PRVs, which can be used to shorten the FDA review time for a new drug down to six months from 10 months, does fluctuate, but typically averages out at somewhere around $100 million. They are awarded to companies who bring new therapies to market for rare paediatric or neglected tropical diseases.

The shortened review time can be very valuable to big pharma companies, especially if they are trying to bring a new product to a competitive market, while for smaller biotechs the sale can provide valuable funding that can be used for operations or invested in other R&D projects, particularly when access to investment funding becomes tougher.

Some have been sold for as little as $67 million, while at the other end of the spectrum prices have gone as high as $338 million at a peak in the resale market in around 2015. Novartis bought another PRV from Mallinckrodt last year for $100 million.

So, why did it get such a good deal this time? The answer is that Pharming was granted the PRV by the FDA after it claimed approval for Joenja (leniolisib), a therapy for activated phosphoinositide 3-kinase delta syndrome (APDS), a rare and progressive primary immunodeficiency disease.

Novartis was developing leniolisib itself for APDS, but had a change of heart following a review of its pipeline priorities in 2019 and decided to license the therapy out to Pharming for $20 million upfront plus milestones and royalties.

The deal also included a clause giving Novartis the right to purchase the PRV from Pharming “for a pre-agreed, contractually defined percentage of the PRV value,” said the Dutch biotech in a statement issued this morning.

It’s not clear, yet, what Novartis may use the PRV for, but in the past it has used one to speed its ill-fated ophthalmic medicine Beovu (brolucizumab) to market - a drug subsequently scuppered by safety issues. Others have been used for multiple sclerosis therapy Kesimpta (ofatumumab) and inflammatory disease therapy Cosentyx (secukinumab).

Drugs currently at the pre-registration stage in Novartis’ portfolio include Kisqali (ribociclib) as an adjuvant therapy for HR-positive, HER2-negative breast cancer, iptacopan for paroxysmal nocturnal haemoglobinuria, and radioligand therapy Pluvicto (lutetium vipivotide tetraxetan) for metastatic castration-resistant prostate cancer.

Pharming’s chief financial officer, Jeroen Wakkerman, said the sale of the PRV will “further strengthen Pharming’s financial position and help fund pipeline developments, including new indications for leniolisib.”