An FDA First: Dompé Shares Experience Of Securing A Commissioner's National Priority Voucher

A conversation with Ahmed Enayetallah, Chief Development Officer, Dompé

In June, the FDA announced a landmark voucher program aimed at reducing application timelines to just one or two months (from the customary year or so applicants usually endure). The program features a “collaborative tumor board style review process” to realize those gains. To be considered, companies must intend to use the voucher for one of a handful of reasons blessed by the FDA, including addressing a U.S. public health crisis, delivering “innovative cures,” and addressing unmet medical needs, among a few others.

Nine voucher recipients were named just weeks ago, with Dompé receiving its voucher for the pursuit of its BLA for an intranasal form of nerve growth factor (NGF), or cenegermin-bkbj, for the treatment of non-arteritic anterior ischemic optic neuropathy (NAION).

In this Q&A, the international pharmaceutical company’s Chief Development Officer Ahmed Enayetallah reveals how the team earned a landmark voucher, as well as how it plans to use the advantage to progress its NGF platform for NAION.

How did the company first hear about the voucher program, and what went through your mind when selected?

When it became public, my first reaction was to think about all the positive and meaningful things that can come out of this. At its core, it's at least in part addressing one of the more chronic problems in drug development, which is the drug development timelines.

NGF actually is a good case in point. NGF was first discovered in the 1950s, but the Nobel Prize for NGF discovery took three to four decades. The first human NGF was approved for neurotrophic keratitis in the U.S. in 2018. For something like that to take 75 years, that's a long, long time.

We have to solve that problem. And so, my first reaction was, “Oh my God, this is exactly the challenge we have at Dompé.” Dompé has a lot of amazing science, but it's that last step that’s the challenge. How do you take the scientific discoveries and realize the therapeutic potential? I welcome any effort in that direction; it is only good for the scientific community and for patients at the end of it.

There's more than one way to receive a voucher — you can be selected by one of the departments within the FDA or you can self-nominate. Which path did Dompé take?

We were selected by an FDA division. To provide context, since I joined Dompé in May 2024, we revamped the entire pipeline, and NGF is at the core of it. We have five ophthalmology programs, three programs in neurology, and then we have other transformative areas. We are going after a real unmet need, indications where nothing is approved or nothing is known to be effective. Non-arteritic anterior ischemic optic neuropathy (NAION), the indication we're talking about for the voucher, is one of those. It's an unmet need and causes loss of vision for many patients, even blindness.

Our program is being enabled by our intranasal route administration. If you think about the first indication approved, neurotrophic keratitis, it's easy to get NGF to the front of the eye, to the surface of the cornea. If you want go to indications impacting the back of the eye, it becomes a challenge for large molecules like NGF to cross the blood-brain barrier. The intranasal administration helps us get around that and unlock that therapeutic potential for NGF. We've been talking to the FDA across all of those programs for some time.

We shared with them that the NGF is not about a single indication. We're not going after symptomatic treatments here and there. We're going after disease modification; we're going after difficult indications.

So, when you have this combination of a molecule like NGF with the science behind it, 75 years of amazing science and discoveries in the NGF space, and the unmet need in neurology and ophthalmology, it's easy to see the potential.

In the middle of those FDA conversations, we were approached by the project manager, and he informed us that the division is considering nominating us for the voucher. We met with them twice, specifically focusing on our development plans, our commitment, and our readiness. A few weeks later, we learned we were actually nominated to receive the voucher. So, it’s amazing news for us. But honestly, if the clinical trials are positive and we can bring something meaningful to patients, it's a huge step.

Under this voucher program, you have to address a national priority. It sounds like yours was the unmet medical need. Is that correct?

I was not part of the process, but I would like to think so. There’s a mix between the unmet need and the potential disease modification from NGF as a platform, not just a single indication. In China, they use it in a dozen or more indications across ophthalmology and neurology. There’s huge potential.

Specifically, there’s an unmet need with NAION. An estimated 6,000 to 7,000 patients per year in the U.S. are getting this kind of loss of vision. And it's scary. In many cases, patients wake up in the morning and, all of a sudden, they just can't see.

In more recent reports, the estimates are actually greater than 35,000 patients a year, and that is linked to a number of things, such as heart disease, metabolic disorders, and being over 50. So, this is an unmet need. It's not being addressed. Nothing is known to be effective. Nothing is approved for it. It's on the rise, and it's one of the most impactful disabilities.

Help me understand where you are in the research process with NGF. How is this voucher going to help you speed things up?

Right now, we are preparing for the launch of our pivotal registrational study. We're planning to start it very soon, and we think that we learned enough from the scientific community and prior experiments, and we're trying to be as efficient as possible based on the data we were able to collect from prior trials, from top KOLs  in the field, and others.

If the study shows us what's needed in terms of efficacy and safety, tolerability, all of that, and if it meets the criteria for approval, then that would be great. Essentially, we're potentially one study away from getting something meaningful and impactful to those patients.

Will the voucher enable more frequent communications with the FDA? And is there any additional effort to ensure you’re using it to the best of your ability?

As excited as we are by the recognition, the scientific value, and the potential impact, it's a huge responsibility. We are taking it seriously in our commitment to leverage the voucher as much as we can. Enhanced communication with the FDA is definitely part of it.

If you go back to drug development and the bottlenecks, it's the uncertainty around what's needed and needs to evolve as the program advances, whether because you have findings you want to discuss with the FDA, or the study design, what's an acceptable package, submission readiness — all of that. If you have enhanced communication and partnership with them, you can hopefully remove some of the bottlenecks.

That can have a significant impact on the total development timeline for a program. If you think about the shortening of the review period, a shorter review usually comes with an earlier rolling submission. It's not that you wait until the end; you start the submission very early. If you have some of the elements ready in terms of manufacturing, such as CMC, you start that rolling submission process. Every time you complete a component or a module, you submit it, and the review is ongoing.

But that's very meaningful if you think about the potential for NGF to address loss of vision. It's a year’s worth of patients who, if this drug is approved, aren’t going to lose their vision. Reducing that time by one year is very significant.

The voucher is good for two years. Does that put any new challenges in front of you or any additional pressures?

No, it's part of the responsibility. We have to be innovative in everything we do, whether in terms of timelines, whether in terms of quality. What you say are challenges are, to us, essentially part of the job. It's more of the heightened sense of responsibility. We do all of that on a day-to-day basis already, but now we have to be more innovative in those areas, maybe more than usual.

What kind of impact does this voucher have on the partners, such as CROs and sites, that you work with?

When the news got out, we were actually approached by many people who said they would love to partner with us. So, we've been getting a lot of those communications. But in terms of site selection and CRO selection, we are already using very high standards. There are things that we just don't compromise on.

I think it is more “How do we innovate scientifically? How do we identify bottlenecks that we believe can be removed and can enhance the process?” And there are meaningful ones, especially if the FDA is open to enhanced communication.

Does this enable you to be creative in your brainstorming and then gut-check with the FDA?

Exactly. We're thinking of this as a partnership and a two-way street. We can't just think about moving faster. We are truly thinking about it as, “Hey, FDA, we think this activity takes a lot of time unnecessarily and here's a way to potentially address it. Can we do that?” Just being able to have that exchange and come up with better ways of doing things will make a difference. In the short term, this will help with programs like NAION, but in the long term, the learnings might be helpful for the entire community in terms of drug development.

It might be too early, but have you gotten any green lights from the FDA on any of your ideas to improve the process?

It is still too early. We probably should be moving even faster than this, but it's been only about two weeks now.

Clinical Leader intends to catch up with the Dompé team later to understand the voucher’s impact as they potentially approach an FDA submission.

About The Expert:

With a background in clinical research and drug development, Ahmed Enayetallah’s career spans several high-profile roles across leading pharmaceutical and biotechnology companies, such as Amgen, Biogen, and Alexion. Ahmed earned his medical degree from the University of Cairo and completed his Ph.D. in pharmacology and toxicology at the University of Connecticut. His early career included significant contributions, where he led efforts in personalized medicine and championed precision medicine initiatives leveraging earlier AI platforms. Later, he led and oversaw multiple successful clinical programs that brought novel therapies to patients with high unmet medical needs. Prior to joining Dompé, he led development at BlueRock Therapeutics and drove the company’s mission to harness the power of cell therapy.