Inaugural Class of Commissioner’s National Priority Voucher Recipients Announced
We previously blogged about the Commissioner’s National Priority Voucher (CNPV), FDA’s new pilot program to expedite the reviews of selected programs that meet certain national priorities. As a reminder, these national priorities were described as including (but not limited to):
- Addressing a U.S. public health crisis
- Delivering more innovative cures for the American people
- Addressing a large unmet medical need
- Onshoring drug development and manufacturing to advance the health interests of Americans and strengthen U.S. supply chain resiliency
- Increasing affordability
The initial press release and other announcements stated that five pilot participants would be selected during the first year, which may be increased in future years. On October 16, FDA announced not five, but nine recipients for the first class of CNPVs. The press release listed the products as below:
- Pergoveris for infertility
- Teplizumab for Type I diabetes
- Cytisinicline for nicotine vaping addiction
- DB-OTO for deafness
- Cenegermin-bkbj for blindness
- RMC-6236 for pancreatic cancer
- Bitopertin for porphyria
- Ketamine for domestic manufacturing of a critical drug for general anesthesia
- Augmentin XR for domestic manufacturing of a common antibiotic
The FDA press release describes the nomination process as two-fold: the sponsor application process that we previously blogged about and nominations by the review divisions, which were each charged with nominating a product they believe qualifies for the program. This latter process means that it is possible that some recipients may not have applied for the CNPV. Even with this larger than expected class of recipients, the press release notes that “[t]he agency anticipates announcing another group of CNPV recipients in the coming months.”
We find the indications for the selected products interesting. While a number have broad descriptions (e.g., infertility, deafness, and blindness), the actual indications are likely to be more narrow. For example, cenegermin-bkbj, the “blindness” drug, is approved as Oxervate to treat neurotrophic keratitis. The sponsor’s pipeline shows that this drug is also in development to treat Persistent Corneal Epithelial Defect (PCED) and optic neuropathies. It’s not entirely clear which of these is “blindness,” but we always support the prioritization of ophthalmologic diseases, with the substantial unmet medical need, by FDA.
The selection of bitopertin for porphyria also stands out; the drug is being investigated for erythropoietic protoporphyria (EPP), an ultra-rare dermatologic condition. As advocates for rare disease patients, we are thrilled to see this inclusion in the inaugural class, as there had been fears that drugs for rare diseases would be overlooked. Rare diseases can occur in any therapeutic area, but dermatology is not where most ultra-rare work takes place.
Another interesting inclusion is teplizumab for Type I diabetes. Teplizumab is already approved within this indication (“to delay the onset of Stage 3 type 1 diabetes in adults and pediatric patients 8 years of age and older with Stage 2 type 1 diabetes”). Presumably this voucher is for a potential indication expansion, as teplizumab is being evaluated in newly diagnosed Stage 3 Type 1 diabetes subjects.
The press release also specifically quotes President Trump about the Pergoveris selection, which appears to be the lone selection related to affordability. As part of an event at the White House on the same day as the CNPV announcement, the maker of Pergoveris, EMD Serono, announced a multifaceted agreement with the U.S. government in which EMD Serono will provide its “complete portfolio of IVF therapies . . . to eligible patients with prescriptions at significantly reduced prices” (referred to in trade press as Most Favored Nation prices). EMD Serono will also participate in the TrumpRx.gov direct purchasing platform when it goes live; additionally, EMD Serono pharmaceutical products and ingredients will be excluded from tariffs, provided it meets certain goals related to onshoring manufacturing and research. This same press release mentions the CNPV receipt to expedite the review of the drug, which is already approved outside the U.S. This outcome is consistent with our speculation that a CNPV based on increasing affordability of drugs would depend on cost-lowering for other, already-approved, drugs because of FDA’s lack of legal authority to make approval decisions based on cost. However, other national priorities may certainly have been implicated here as well.
There are also two selections related to the national priority of onshoring manufacturing: one for a “critical drug” (ketamine) and one for a “common” drug (Augmentin XR). At least one of these was granted to a CDMO; presumably the CDMO would need to submit the marketing application itself, as a CNPV cannot be transferred (unless that changes as well).
For other selections, it is not quite as easy to identify just one national priority, as there is substantial overlap between concepts such as addressing a public health crisis, delivering more innovative cures, and addressing a large unmet medical need. It is possible that all of the selections had arguments to meet multiple priorities.
Regardless, we will be paying close attention to see if this inaugural class graduates on time, and we look forward to seeing what comes next!