FDA Direct: These 9 Drugs Were Awarded National Priority Vouchers

Hey, folks, we're doing this.

As the team here knows,
I don't like to do mic checks.

I like to get right into it.

And today I'm getting right into it
with Doctor Mallika Mundkur,

who is one of our great physicians
in the office of the CMO

and the lead for the National Priority
Review Voucher Program.

So Mallika, great to see you
and thanks for doing this.

Thank you so much, Marty.

Real quick,
just so the audience can get to know you.

Yeah.
Tell them a little bit about yourself.

Specifically, are you afraid

of bears or hawks?

So I have a funny story about bears.

Oh, boy. Yeah.
So I'm not. I'm not afraid of either.

Actually, I should be, but,
we were at Yellowstone National Park

a couple, I think,

months after I got married, and,

I had to get those pictures.

You know, the wildlife. It's everywhere.

So. In front of a cub underneath the mother?

Well, I did get out of the car
to try to take a picture of a baby.

Okay, that's one thing in the manual
you're not supposed to.

Yeah, I was, I was.

Don't judge me.

Yeah, I have made better decisions
since then, but I did.

I was encouraged to get back into the car
very quickly,

and I did, I did,
and I'm here to tell the tale.

So. You're fearless.
That's what we like at the FDA.

People who are fearless.
Got to get that picture so. Good.

Well, you can share it with us at some point.

Where'd you go to medical school?

University of Massachusetts. Okay.

Is that a state in New England?

Yes. And college before that. Harvard.

Okay. And,

residency went to UAB.

Roll tide.

Know my college football.

University of Alabama in Birmingham. Yes.

Great school.

Yes. Thank you. Great.

All right.

Well, with that, now, you know Mallika
here, our wonderful colleague at the FDA,

you two can work at the FDA
and be a Mallika someday.

If you'd like to apply to work here.

We are constantly in our hiring mode, so,

tell us about the National Priority

Review Program that you helped craft.

And just sort of the overall, concept
behind it.

Sure. So,

in a nutshell, I would say it is,

innovation, right?

Innovation and our review processes.

And so I've been at FDA
for eight and a half years

and have to really commend you
for the really

the extent of innovation
that you're piloting,

even in the very short time
that you've already been here.

So it's been incredible.

This program is really,

a forced

innovation and reexamining
our review processes from soup to nuts.

And, you know, what's what do we
what do we really need to do?

How fast can we do it?

Are there ways to modify
how fast we do a review

based on how important we think it is
for the country?

So kind of thinking about the program
and how it, you know,

the broader structure, the usual timeline
for our reviews is, is ten months.

Historically, priority
reviews is six months.

Now, this program aims to have the review
done in a period of to months.

So that is ultra accelerate,
ultra accelerated review.

And when I first heard about,
you know, this this concept,

it sounded wild, like I'm going to say
like it sounded wild.

But as we got into
thinking about this more,

we learned that,

you know, there are components that

make this framework feasible

and not only feasible, it makes sense

to, to do this
when when you have a national priority.

So any of the national priorities
I think you identified for us

and with with others, other leaders
in the agency, I would love to hear

about. A large unmet public health need.

Yeah.

Domestication of manufacturing

as a national security issue,

equalizing the price of drugs

with other OECD countries
to make drugs more affordable.

And also therefore accessible.

And, companies

that are, innovating in a space
where there's a big need

and there's otherwise
very little attention,

areas of medicine
where we have not been giving

a lot of attention that does work,
but deserves a lot of attention. So,

now I, I love this idea.

We got to try new things.
We have to innovate.

We have to be creative.

We have to do things differently.

And if you look at the process
of how we approve a drug

right now at the FDA, I mean,
first of all, people could be involved.

It gets farmed out to multiple offices.

There's no real incentive for you
to get your review done sooner

if you are able to get it done sooner,
because the other offices

will have right up until that
to month point, to get their review.

And so if you're getting your reviews done
in two months,

you're not really increasing
the speed of the overall product.

And we like speed, but we don't like
cutting any corners on safety.

So that is our number one

priority throughout this is
there are no corners cut on safety.

It's the same people, same decisions.

We reserve the right to take more time
if we believe it needs more time.

But the goal is to get these decisions out
in a week or two by reducing idle time,

sorry, a month or two by reducing our
maybe a week or two, who knows?

By reducing the idle time
and reducing the time

where applications are just sitting
around not being reviewed.

So with that,

shall we announce the first recipients

of the National Priority
Review Voucher Program? And,

I'm thinking this will be the first batch.

And then in a couple of weeks,
we'll announce the second batch.

Because it was a big group,
there was some tough decisions.

You can maybe share

how we made the decision
and where the nominations came from.

Yeah. So,

so I do want to say that

a lot of work has gone
into getting us to this point,

you know, which not just folks,
you know, like, like yourself

who are very busy and spread,
you know, across a lot of obligations.

But other leaders and, you know, see the,
the review divisions,

the center directors,
it's been a lot of minds have gone into it

and it's really exciting
to be at this point.

It is kind of cool.

It is.

And I got to be honest with you,
I thought some people would say

here, that's a crazy idea.

We're not playing ball.

But instead what I got was,
I love the concept.

I'd love to help operationalize it.

And so a bunch of people came up to me
and they said, not only,

hey, here's an idea on how we can reduce
some of the idle time, but they also said,

I'm, I've got a medication in my portfolio

that would be a perfect candidate
for this.

And it got me thinking, hey,
instead of us picking the medications,

how about let's just have the divisions
nominate the products that they think

would be a perfect fit for this? Yeah.

And that was that was a great I think,

evolution that

we, you know, during this
the early phase of this program is to, to

not only, you know, engage the, the staff
here and have them part of the process,

which is the way to make this sustainable
and the change sustainable.

But they are also
the subject matter experts.

So they really understand the field
and which, you know, which areas

might be most appropriate for this.

So. Yeah. That was that was a great
you know, that was a great interview.

Well,
I think if we were to just said on day

one, hey, we're doing this, then
we would have had all kinds of issues.

But by socializing it and, you know,
I get a lot of questions,

hey, when is it going to roll out?

Well, we've been here about six months.

I've been here about six months.

You've been here eight years.
Eight and a half. Eight and a half.

With a little break in there
to take pictures of baby cubs and then,

by taking those six months
to think about it, socialize it,

run it by the people,
find out which medications

they think are the perfect candidates
for this, or at least initial candidates.

Because if you're going to do something
on a pilot level,

you got to start with some group.

And sure, we could have just
randomly chosen medications,

but we thought, let's choose medications
in line with our national priority goals.

So I'm pretty happy about the the list,
at least the full list.

This is just going to be the first batch
that we talk about today,

because we only have so much time
to shoot this podcast.

So let's go through the initial list
if you want to run through it.

Yeah, sure. So

so the first one,

this is and this is no, in
no particular order, by the way, these are

these are all exciting.
They're all important.

This one Cytisinicline, is a product
that's proposed

for the treatment of vaping cessation
or e-cigarette.

E-cigarette cessation.

So we don't have anything
FDA approved for this, right now.

And this is a huge problem that you've
you've already talked about,

you know, the vaping problem
in this country.

And. Yeah. This is going to this going
to address a very important, unmet need.

If this goes, you know,
if this goes, as we hope.

Cytisinicline.
I don't know if I'm saying that correctly.

Cytisinicline.
That's kind of how I'm reading it.

Yeah.

You don't have to learn how to pronounce
it out there in the world,

but we, want to, first of all,

we don't know what we're going to decide
on any of these drugs, right?

We reserve the right.

That's right.

We're going to get a decision.
How quickly.

But it's not a guaranteed approval.
How quickly?

And so the review team, led by the subject
matter experts,

that is the primary review team
within their division.

If they say, hey,
we're just not on board with this, then

you know what may not get approved.

So, there's a committee
that you're heading up

that is a multidisciplinary group
of scientists and doctors

that are going to be reviewing
these medications.

But this is interesting
because million Americans

use tobacco or nicotine products,

and there's nothing really approved
specifically for vaping cessation.

Yeah.

So this could be a potentially promising.

Absolutely.

So I think opportunities to innovate
in that space and in terms of,

treating nicotine dependance
now that it's a problem for our youth,

is, is finding, finding treatments that
that will, that they will adhere to.

Right. So that that's a challenge.

So I think this is a
this is a really, important,

product for us to try to accelerate.

Yeah, potentially
promising large unmet public health need.

I think it's that category perfectly.

Yeah. Great.

Absolutely.

Okay.

Next on the list okay.

This the pronunciation.

Don't, don't hold me to this. Cenegermin.

Yeah that's how I would pronounce it
okay. Yes.

-bkbj whatever. Yes. Yeah.

So, this is, it's an interesting,

very interesting product in my opinion.

Recombinant human nerve growth factor.

This is already approved
for another condition for the treatment

of something called neurotrophic
keratitis, an ocular condition.

But here the proposed formulation is it's
going to be intranasal. And,

the, the
hope is that it's going to sort of promote

return of vision
in, for, for this condition called NAION.

Nonarteritic

Anterior Ischemic
Optic Neuropathy. One of the most common

forms of Optic Neuro-.
The most common form of Optic Neuropathy.

Yes. So this this could potentially help
a lot of people.

A, you know, blindness is,

a terrible thing.

And if you look at surveys
after the fear of death,

the second most common fear or third
most common fear that people have,

depending on the survey, is blindness,
and it is debilitating.

So, look, I hope this can potentially help
a lot of people.

Yeah. Yeah.

Okay. Next on the list.

All right.

So Teplizumab. Teplizumab.

Yeah.

Teplizumab, for type one diabetes,
which I know is also it's

a is a, a condition of interest right now
type one diabetes.

It's the pathophysiology.

It's an autoimmune disease

effectively where where the body is sort
of attacking its own pancreas.

And this product is it's an antibody,

that aims to sort of decrease
that autoimmune activity

and delay the progression of,

destruction of the pancreas
by by the autoimmune process.

Yeah. Specifically the CDcells.

So those CDcells are destructive
to the beta cell.

And so this is an anti CD
monoclonal antibody,

which is has shown
promise in other settings.

And I think phase one and phase
two of type one diabetes.

So this is up for being evaluated

now for a common phase, phase 

And gosh, if it can help reduce the number

of insulin shots
that somebody needs, that'd be great.

Right.

And the times that they're in the ER
with DKA or

other complications of type
one diabetes is particularly

as it progresses to those later stages, is
is really a terrible disease.

So this prevention is is the key.

So hopefully
we can see something happen here.

I’d love it if it could help my cousin
with type one diabetes.

He's been dealing with it for a long time.

But we're going to go into this
or the committee will go into this

very objectively.

We will. Trying to review that data.

But the division that,

nominated this,

nominated it with some enthusiasm.

Yes.

Next medication.

All right.

This one is really
I am very excited about this one.

It's it's, a gene therapy product.

It's their own gene or only gene therapy
product in this first batch, DB-OTO,

there was a publication that just came out
in NE JoM a few days ago.

Yeah, I think I saw that. Yeah.

Describing
some of the clinical results. So,

they in that study, it was a,

it was a first in human open label
single group study, that demonstrated

improved hearing in % of treated
children with congenital genetic deafness.

So that's that's the
the area that this treats.

So and three actually achieved
normal hearing sensitivity.

I'm not I don't work for the company.

But this did that did excite me.

You know
these are pretty remarkable results.

So safety is another question.

I, I care a lot about safety.

And I think that is a piece
that we need to learn more

about. Always a question
with gene therapy in particular.

But I saw that
New England Journal sort of 

subgroup of the couple.

Maybe it was three.

I guess you're telling me,

people who had their hearing

restored to normal and gosh,
it just it would you how can you not

look at that and say,
we need to get a decision out promptly?

You need to. Right.

Not approve it quickly.

We just need to get a decision out
promptly

using the standard scientific process,
cutting any wasted time.

Any idle time.

Right. But, pretty interesting mechanism.

Yeah.

Now it's only for like 

to % of children with,

congenital deafness

actually have this gene defect. Right.

That's right.

So this is very rare.

It's a very rare disease, but has the
potential, you know, with this treatment

to change the course of that,
that child's life.

So in that way pretty exciting.

But long term safety and effectiveness
that that's definitely something that

will be interesting to
to learn more about here.

And we're hearing
the company may make it available

either at an extremely low price
or for free, which of course

is part of a national priority
to make drugs more affordable.

So we would love that.

All right, next product.

So I'll maybe mention two,
but we'll start with the first one.

So ketamine.

Ketamine is, dissociative anesthetic.

Many off label uses that have taken hold
in, in the country.

But, the proposed indication for, for
this product is anesthesia,

but the priority is domestic
manufacturing, which is, you know,

certainly there's
executive orders out there that really,

emphasize the importance of building
supply chain resilience.

Bring your manufacturing
to the United States,

and there will be a treasure
trove of benefits, number

one of which is national security
for the American people.

So this is an America

first priority is bringing manufacturing
to the United States.

A lot of companies are saying yes to it.

We've offered to help with the process.

We have a pre-check program now,
among other programs, to help companies

that want to set up their, facilities
in the United States

with more of a partnership
with the FDA to get the,

approvals done
early for the manufacturing part.

And the other one. Yeah.

So the the other one, Augmentin.

Oh, by the way, ketamine. What?
Who's the sponsor?

Flow. Flow.

Okay, probably
good for us to mention the sponsor.

Yes. Great.

And also with the next one.

So the the next one. Augmentin.

Same priority is domestic
manufacturing supply chain resilience.

And the company is US antibiotics.

So love the first two letters US.

Yes love it. Yes. Me too.

million prescriptions
a year are written

for antibiotics.

It's not just Augmentin, No, 

That's antibiotics in general. Yep.

Probably should be million.

I was thinking,
I was thinking the same thing.

I'm like, should I say

Say whatever you want.

We prescribe too much, but we want it
when we want to have it when we need it.

Right? And we want it to work.

Antibiotics save lives. Yeah.

And they are a critical tool.

One of the biggest advancements in modern
medicine, in the history of medicine.

But we are at risk of going backwards
to the time

before the advent of antibiotics
because of the overuse driving resistance.

And so that is a big problem.

Study after study has shown roughly
half of outpatient antibiotics

are entirely unnecessary
or should not be prescribed.

We all know what's going on, right?

It's a consumerist culture.

It's easier just to give a prescription
to somebody with a viral upper

respiratory infection
than to say, this really does not appear,

to be, a fit for antibiotics.

Call me tomorrow if you happen to notice
symptoms of a bacterial infection.

But it is a pill popping culture
in many aspects by the way,

the United States may not be the worst

we're hearing overseas
where you don't even need a prescription.

It's like given out, like can- candy.

I mean. That's true.

You take it for headaches
in some countries inappropriately,

they just see it as,
oh, it's this incredible magical thing

and it is magical.

Go to the pharmacy in India and and buy,
buy this stuff without prescription.

Yeah.

Literally street vendors
and and all over the world.

Yeah.

So antibiotics save lives.

We want people to take them.

They do amazing things for people
with bacterial infections.

We also have an overuse,

we have got an underuse problem
and an overuse problem.

But the overuse problem
dominates the marketplace,

the overuse problem in general,
but also with antibiotics rates.

Amen to that, sister.

Okay, so Augmentin is a candidate.

Congratulations to that company.

And thank you for making it
in the United States of America.

Okay. Next product. Okay.

This one is also,

really important in,

in its alignment
with our national priorities.

So the product is Pergoveris.
and the company's Merck Serono.

And maybe you could talk a little bit
about that.

Yeah. It's for infertility.

And look at addressing infertility is a
massive priority in this administration.

Infertility is a big problem.

And it is a problem that we have
to address in a multipronged fashion.

We have to address the root causes
and the insulin resistance.

Why are sperm counts going down?

Why are we seeing more polycystic
ovarian, disease or syndromes?

Why are we

not talking about the root cause

and preventing when somebody comes in
with persisting ovarian disease?

There are actually dietary things
that can,

help with that condition.

But we really are not taught in
medical school how to start talking

about food.

Somebody comes in with polycystic
ovarian disease.

So infertility is a big issue.

And this is one piece of it.

This is an aid, medication for women
undergoing IVF procedures.

It's approved in other countries,

and it's in line with President Trump's
executive order to address infertility.

Yeah.

So I think,

this one,
this one is this one is important.

Very important.

Yeah.

Next medication is for...

Erythropoietic Protoporphyria.

or vampire syndrome or is that?
That sounds better.

Is that politically incorrect?
or sounds like it would be.

I think we're allowed to say vampires.

Okay, well,
that's the historic name for it.

The lawyers are taking some

antacid right now
because. They are looking it up right now.

Are we allowed to say this?
They just see us here on the set,

They know I'm I'm going to just say whatever.

Yeah.

So, it is a small molecule
and it's for these children

where when they go out in the light
in the daytime,

it starts affecting their metabolism.

Porphyria can result in some,

liver toxicity from the byproducts of,

what gets, converted because of the,

light exposure
or what we call photo toxic.

So the division that nominated this again,
I thought this would be a good fit.

And so.

Sounds potentially promising. Right.

Rare disease, but incredibly,

you know, it
it it influences the child's life.

If you can't go outside
as a child to talk about,

you know, the social consequences
and the emotional consequences long term.

So this it is a game changing,
potentially a game changing product.

The product name, by the way, is DISC 

I don't know if that's the final name,
Bitopertin. Bitopertin.

And it's the company
I believe is DISC. DISC Medicine.

Okay.

I, I'm glad you mentioned
rare diseases because we're committed to

doing everything we can for rare diseases.

This National Priority
Review Program is not designed

to encompass, the Pediatric Priority
Review Program is designed to complement.

I have been on the Hill advocating
for the Pediatric Priority Review

Voucher Program, and I'm optimistic
that will get renewed by Congress.

So we are very committed to that program.

Yeah.

Which is distinct from this one. Yes.

Let's do one more. Okay.

And then we'll do batch
two in a couple of weeks maybe.

Yeah, that sounds good.

This one

is, a product for pancreatic cancer,
which is a KRAS inhibitor.

Yes. We,

you know, pancreatic cancer,
that this is certainly a large unmet need.

We don't we don't have, we don't have
great treatments out there for this yet.

So if you want to talk a little about. Sure.

So maybe to people a year,

get diagnosed with pancreatic cancer.

Yeah.

For a subset, a small fraction of them
will have a certain genetic profile

that makes them a candidate for this KRAS
inhibitor, at least the candidate

for the trial in the trial criteria,

the medication is RMC-

And the oncology division thought

this is something
that is potentially promising,

and they're enthusiastic about
having this part of our national priority

review. Pancreatic cancer,
one of the most common cancers

in the United States in the world of GI
cancer, one of the most common GI cancers.

GI cancers, of course,
are not seeing the reductions

that other cancer types are seeing in,
all of medicine.

So, we'll see.

This was Revolution Medicines? Revolution
Medicine, this sponsor.

Yeah, yeah. Okay.

So we'd like to, hopefully
we can see some promising results.

Look, we are going into the pipeline
talking to the reviewers, asking them,

what do you think
may potentially look amazing?

Yeah.

And so we are not taking a receive
only mode.

We are going into, the divisions
asking them,

tell us what you think is potentially

amazing,

potentially promising.
Transformative. Transformative.

And then let's reach out to the companies
and see maybe we can enhance

the communications, we can, reduce
the idle time, we can cut the red tape,

and we can do things like this
pilot program to meet our broader goal

of more cures and meaningful treatments
for the American public,

and healthier food for children,
which is separate from this discussion.

So great. Any parting thoughts,

Mallika? I am excited about the innovation
that's happening.

So just, you know,
thank you for for the ideas.

And, look forward
to seeing kind of what happens next.

Thank you for leading this effort.

We I would we would not be able to do
so many of the things we're doing.

Were not for amazing people like yourself
that have been here at the FDA,

know the FDA, our amazing
physicians and scientists

and have responded and said, yes,
this looks amazing.

I want to be a part of it.
So thanks for leading this.

Thank you. Thanks.

All right folks. And to the reviewers too.

Yes. The many reviewers.

I wish we could just rotate them
through here and give them some credit.

You know, they work so hard.

Amazing people.

Maybe you'll see some of them

walking around in the background,
but they deserve more airtime than that.

But all right, folks, thanks

So much. And we'll do it again.