The US Food and Drug Administration (FDA) accepted for review the New Drug Application (NDA) for Sanofi’s investigational type 2 diabetes treatment on Monday and granted the use of another Priority Review Voucher (PRV), which speeds up FDA’s decision by four months.
In redeeming the voucher, Sanofi had to pay a $2.7 million fee in addition to the standard new drug filing fee of $2.4 million.
Retrophin sold the PRV to Sanofi last May for one of the highest amounts ever: $245 million (AbbVie bought one in August for $350 million). The sale to Sanofi came after Retrophin obtained the voucher following the 17 March 2015 approval of Asklepion Pharmaceuticals' drug Cholbam (cholic acid), which is the first FDA-approved treatment for pediatric and adult patients with bile acid synthesis disorders due to single enzyme defects, and for patients with peroxisomal disorders. Asklepion transferred the PRV to Retrophin, which had licensed the rights to the voucher under an earlier agreement.
This is Sanofi’s second PRV used. The first was purchased from BioMarin for $67.5 million and used to speed the approval of a PCSK9 cholesterol drug known as Praluent (alirocumab), which won FDA approval last July. The first rare pediatric disease PRV was given to BioMarin in February 2014 after its rare disease drug Vimizim was granted FDA approval.
How the Vouchers Work
As the Focusexplainer on PRVs details, the two PRV programs include: one for treatments that address a list of more than 20 neglected tropical diseases and another for rare pediatric diseases affecting fewer than 200,000 people, more than half of whom have to be under the age of 18.
Nine PRVs have been awarded to date: six for rare pediatric treatments and three for tropical diseases. The list of tropical diseases has been periodically updated by both Congress and FDA, and currently there’s a bill in the Senate and one in the House to add Zika to the list of tropical diseases earning a drug developer a PRV.
Both types of vouchers can be bought and sold an unlimited number of times, and the price for PRVs has been steadily increasing as more vouchers come to market.
Despite the thriving market for PRVs, some experts have questioned who it is that's done the bulk of the research to develop the drug that allows a PRV to be obtained. The rare pediatric disease priority review voucher program is scheduled to end in September 2016 unless Congress re-authorizes it.
Four of nine PRVs have now been used to date, though only one drug linked to a voucher has won approval (Praluent), while another used by Novartis for a supplemental Biologics License Application (sBLA) in 2011 was rejected by FDA. Gilead previously paid $125 million for a voucher and announced in July that it’s using it to speed the review of its potential HIV treatment.
Type 2 Diabetes
An FDA decision on Sanofi’s fixed-ratio combination of basal insulin glargine 100 Units/mL and GLP-1 receptor agonist lixisenatide is anticipated in August 2016, a four-month shorter review timeline than if the voucher was not used.
This NDA is based on data from two Phase III studies, which enrolled more than 1,900 patients, and the company says both studies met their primary endpoints and will be presented at a medical congress in 2016. Preparations are on track for Sanofi to submit for EU approval in March.