FDA hands out first national priority vouchers to nine firms

The FDA has named the first products to take advantage of its recently announced Commissioner's national priority voucher (CNPV) programme, a fast track for medicines that could deliver a decision on approval within one to two months.

The pilot scheme – for new medicines deemed to be of national priority as they could deliver breakthrough therapies to patients who urgently need them – will be put through its paces in reviews of nine medicines, including Merck KGaA's fertility drug Pergoveris, which has also just announced a pricing deal for fertility medicines with the Trump administration.

The others include a new indication for Sanofi's Tzield (teplizumab) in newly-diagnosed type 1 diabetes, Regeneron's DB-OTO gene therapy for a form of congenital deafness, Revolution Medicines' pancreatic cancer candidate RMC-6236, Disc Medicine's bitopertin for porphyria, Achieve Life Sciences' cytisinicline for nicotine vaping addiction, and Dompe's cenegermin for non-arteritic anterior ischemic optic neuropathy (NAION), a form of blindness.

The final two are designed to address the need to ramp up domestic US production of essential medicines and reduce reliance on imports, specifically general anaesthetic ketamine and an extended-release formulation of the antibiotic combination amoxicillin/clavulanate (Augmentin XR).

The CNPV programme, first revealed by FDA Commissioner Marty Makary in June, is designed to address major national priorities, such as meeting a large unmet medical need, reducing downstream health care use, addressing a public health crisis, boosting domestic manufacturing, or increasing medication affordability with most favoured nation (MFN) pricing.

The time saving over the usual 10-12 month review timeline will be delivered by bringing together FDA experts from different disciplines to carry out the review simultaneously, rather than using the standard system of a drug application being sent to numerous FDA offices sequentially, culminating in a one-day meeting. It will also allow greater interaction between the sponsor and the regulator, and sponsors can submit some parts of the data package to the FDA in advance.

"One of our core goals is to deliver more cures and meaningful treatments – especially ones that have an outsized impact on our most pressing national priorities," Makary said in a statement.

"We must modernise the review process and try new approaches to meet the needs of the American people."

The FDA said it anticipates announcing another group of CNPV recipients "in the coming months." There has been speculation that the next tranche could include Eli Lilly's oral GLP-1 agonist orforglipron for weight loss and diabetes.