The FDA has selected Dompé to participate in its Commissioner’s National Priority Voucher (CNPV) pilot program, representing a major win for the biopharmaceutical company and its investigational therapeutic for non-arteric anterior ischemic optic neuropathy (NAION).
To note: Dompé is just one of nine recipients chosen—and the only company in the ophthalmic space—for this inaugural program (but more on that later).
Let’s begin with Dompé.
Operating its international business out of Italy—with U.S. headquarters in San Mateo, California—the privately-held Dompé is active in all areas of the pharmaceutical value chain.
From the research and development phase to the production and commercialization phases, the biopharma giant boasts a pipeline targeting three therapeutic areas: the eye, the brain, and pain.
And zeroing in on the eye …
Dompé’s ophthalmic involvement began with developing a treatment for neuropathic keratitis (NK) based on nerve growth factor (NGF) research and a biotechnological process.
- To note: That drug has since been approved by the FDA (in 2018) and marketed as Oxervate (cenegermin-bkbj) ophthalmic solution, 0.002% (20 mcg/mL).
- Its claim to fame: This prescription eye drop is the:
- First topical biologic FDA-approved treatment for NK
- First recombinant human nerve NGF (rhNGF)-based therapy for NK.
- Its claim to fame: This prescription eye drop is the:
Hold up: Refresh me on NGF.
NGF is essentially an endogenous protein that plays a role in neuron differentiation and maintenance.
- During its discovery, neurobiologists determined that NGF is also critically involved in the proliferation, differentiation, and survival of sympathetic and sensory nerves—as well as pain perception.
And in recent research?
NGF’s therapeutic properties have been found to benefit human cutaneous and corneal ulcers, pressure ulcers, glaucoma, maculopathy, diabetic retinopathy (DR), retinitis pigmentosa (RP), and other ophthalmic diseases.
- As for Dompé: The company acquired the rights to produce and market NGF-based treatments in 2010.
Next: This NAION therapeutic.
That would be the NGF formulation of cenergermin-bkbj, intended to be intranasally-administered as a patented, non-invasive delivery method for treating NAION—which currently has no approved treatments to reverse vision loss or prevent worsening in diseased patients.
- Notably, this therapeutic differs from Oxervate in its intranasal rather than topical delivery method.
As for its purpose: The intranasal NGF aims to preserve and improve visual function in patients with vision loss secondary to NAION by bypassing the blood-brain barrier.
And how far along in the development stage is it?
According to Dompé, plans are underway for a registrational pivotal trial program in over 130 clinical stages across 16 countries.
- No further details on this were released.
Duly noted. Now tell me about this FDA program Dompé is participating in.
Announced earlier this year, the CNPV pilot program was launched to expedite the development and review of prescription drugs and biological products that have significant potential to address critical U.S. national priorities.
- A few examples of these priorities include meeting a large unmet medical need (such as a rare disease like NAION) or addressing a public health crisis.
- See here for eligibility criteria.
And what are the advantages, if selected?
Recipient companies are given the opportunity to reduce the FDA’s standard application review time for investigational candidates from 10 to 12 months to just 1 to 2 months.
- How it’s doing this: Via a collaborative, multidisciplinary review process to accelerate approvals that involves a one-day “tumor board style” meeting.
Other program benefits include receiving a voucher that provides:
- Enhanced communication with the FDA
- A rolling review to facilitate a faster timeline
- Potential for accelerated approval (if applicable requirements are met)
- Maintenance of the FDA’s safety and efficacy standards
Nice! So what’s the plan for Dompé’s NAION candidate?
Following that aforementioned planned clinical program, the company intends to use its voucher for a Biologics License Application (BLA).
Stay tuned in the coming months for more details on clinical trial specifics and timeframes.