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Sales and Marketing

1rem

Approval was based on results from the MANDARA Phase III trial, which demonstrated the superiority of Fansenra over Nucala in patients with eosinophilic granulomatosis with polyangiitis. 

FDA Approves Fasenra for Eosinophilic Granulomatosis with Polyangiitis 

Approval was based on data from the IND.227/KEYNOTE-483 trial, which demonstrated a 21% reduction in the risk of death when Keytruda was added to chemotherapy in patients with unresectable advanced or metastatic malignant pleural mesothelioma. 

FDA Approves Keytruda Plus Pemetrexed, Platinum Chemotherapy for Unresectable Advanced or Metastatic Malignant Pleural Mesothelioma

Results from the ADvocate 1, ADvocate 2, and ADhere clinical trials found that patients treated with Ebglyss 38% of patients with moderate-to-severe atopic dermatitis achieved clear or almost-clear skin after 16 weeks of treatment. 

FDA Approves Ebglyss for Moderate-to-Severe Atopic Dermatitis 

Ocrevus Zunovo is the first and only twice-yearly, 10-minute subcutaneous injection for treating both relapsing multiple sclerosis and primary-progressive multiple sclerosis. 

FDA Approves Ocrevus Zunovo for Relapsing, Primary-Progressive Multiple Sclerosis 

Subcutaneous Tecentriq Hybreza will provide patients and physicians increased flexibility for administration of the immunotherapy across approved indications for Tecentriq, including non–small cell lung cancer, small cell lung cancer, and hepatocellular carcinoma. 

FDA Approves Subcutaneous Tecentriq Hybreza for All Adult Indications of IV Form of Tecentriq

Citius Lymphir BLA

PE: The FDA recently accepted the BLA for Lymphir as a treatment for patients with relapsed/refractory CTCL after at least one prior systemic therapy. How has the company addressed the concerns raised by the FDA in the complete response letter issued in July 2023?

That occurred last July. There was a test on the finished product that we passed, but the validation procedure hadn’t been completed. It was going to run several months past the PDUFA date, and we worked on that. That application was actually worked on a lot by Eisai Pharmaceuticals, who we have the license with. I think everybody thought that the agency would give us an approval condition upon completing that validation test and not go to market until that could happen, but the agency chose to go the other way. So, we received the Complete Response Letter, and we finished the validation procedure. We then resubmitted it to them and they accepted. We have a PDUFA date of August 13th, 2024. We’ve engaged in a process with the agency and hopefully, we'll see an approval in at that time frame. Shortly after, we’ll go to the market. It's a small market throwing 3000 patients out there, so this is an orphan drug, and it's not going to require a giant sales force to cover the market.

PE: Are there any other investigational products in the Citius pipeline that you wish to highlight?

First of all, there are two INDs out for Lymphir, and one is at the University of Pittsburgh. The investigator is an OBGYN/oncologist and he’s looking at a combination of Lymphir and Keytruda. This is because Lymphir has a very unique mechanism of action. It lowers the T-regs in the microenvironment of a cancer cell, which has a tremendous impact when it comes to PD1 inhibitors such as Keytruda, because the overall efficacy of a PD1 inhibitor is impacted by a higher number of T-regs. The high the number, the less efficacy. If you can bring it down, you'll have greater effort.

We had an animal trial that was published last winter in Frontiers Immunology that showed that the combination of our drug with the PD1 inhibitor was better than monotherapy. We’re going to see that there’s a real scientific rationale for this combo.

The way that he’s administering it, the patient starts out on Lymphir, they get their T-regs lowered, and then the patient is given Keytruda or a PD1 inhibitor. We’re very excited about it but we don’t know yet, because it’s early. The investigator has indicated that he’s applied to present a paper at a conference in November, so we should have some data starting in the fall. We'll start to see what the impact of that combo is.

The other one is early on, and it’s with the University of Minnesota. It combines Lymphir with Cart-T another well-known therapy for cancer. It’s really interesting for us to see this happen, because we truly can expand the number of lifesaving situations for the company.

Videos

In an interview with Pharm Exec Associate Editor Don Tracy, Leonard Mazur, Co-founder, CEO, Citius Pharmaceuticals, offers an update on the recently accepted Biologics License Application for Lymphir by the FDA and other candidates currently in the Citius pipeline. 

Video Series

Citius Pharmaceuticals Co-Founder and CEO Discusses the Recently Accepted Biologics License Application of Lymphir for the Treatment of Relapsed/Refractory CTCL

 Co-founder and executive director of the Alliance for a Stronger FDA

PharmExec Video Interview - Steven Grossman, Co-founder and executive director of the Alliance for a Stronger FDA

What prompted the founding of the Alliance for a stronger FDA and why is it important?

In the mid 2000s, FDA was going through a period of recalls of controversies, food, medical products, other areas. And a group of us, a number of us started to worry that Congress would react the way it usually does to agencies that are in trouble. And that is, they cut the funding until they perceive they have the agency's attention. FDA was already extremely underfunded. And going through a cycle of FDA, FDA being further downgraded in terms of resources was just unthinkable.

So, a group of us came together, the original impetus was from the patient community, and we moved forward on this, what was really an experiment. And the founding principles were really simple, but they were to the point, they would always there had been organizations along the way, food, money, and pharmaceutical money. And we thought that was the wrong approach that FDA needed funding at the global level, every part of it needed more funding, and that it interacts, interacts in ways that people tend to forget that is that when there's trouble on the food safety side, the medical product side gets dinged. And vice versa, the American people don't look at FDA is two agencies, one of which is having troubles and the other which is fully respected. The agency rises and falls. So medical products, for instance, which is your area, it's affected by how food is doing. And so, we came together and said, look, rising tide is going to lift all boats, the agency needs resources and everything that does to give you a sense of how bad it had become, FDA got as many as many agencies did, got a post 9/11 bump in fiscal year 2003. And by the end of fiscal year 2005, the entirety of it was born and none of it had been added to the base of the agency, even though the monies are paid for ongoing functions. So, we came together, we were determined to be multi the multi stakeholder to represent all viewpoints that work for the agency as a whole. That work that's how we got together.

We've accomplished a lot in the year since notably, we've been part of a doubling of the taxpayer funded portion of the agency, which is going from 1.6 billion to when we started 2353 point 5 billion in fiscal year 2023. The in this in parallel user fees have also gone up. And the agency has also acquired the tobacco function. So, the agency itself has grown enormously. But all the other monies, the commanded particularly through medical product user fees, are acquired, if you will, for set purposes. They're broad enough purposes. We think, you know, our group supports user fees, but our focus is the money that taxpayers are paying, because that's the part that the appropriations committees look at. It's the part that determines whether or not FDA has the base and the resources to meet all the needs. They're being it's being asked to address.

What does the Alliance for a Stronger FDA do?

The alliance is, as I said, it's multi stakeholder, we have patient, patient groups, we have consumer groups with health societies like the American Heart Association, we have trade groups, and we have industry. And it is all by itself a testament to the agency. There is no other federal regulatory agency in which consumers and industry go in together to ask more for more resources for their regulator, it's why FDA is very special. But it's not a message that sells itself, it has to be brought to people we spent a lot of time dealing with, really FDA covers that, that and that and that the one of the comparisons I make is to the Social Security Administration, which is significantly larger than FDA like massively bigger, they do about seven or eight things. And they do them millions of times every year. It's a very good agency, by the way, but they only do seven or eight things. Maybe somebody who knows the agency better could decide it's nine. But it's not very much. It's not a large number. And I could start now talking about all the things FDA is responsible for. And two hours from now, I wouldn't have exhausted the list. And that's, again, a message that can be delivered, it can be understood, but it isn't apparent, unless it's brought to your attention.

The alliance has two missions, and the mission was met meet with each other. And I'll explain why in a minute. The first responsibility is to make sure the Congress understands what FDA does, why it needs more resources every year, why? It's closer to being, say, the equivalent of air traffic controllers, they are vital to American life and commerce. And you wouldn't think of cutting back or deciding that, you know, we had 10 controllers in, in, in national airport that in Washington, we probably could just do the job with four, you'd never think to do that. And FDA sort of the same way. It's vital to the American people. It's assuring quality. It's bringing its gold standard of regulation to so many different things. And so, our first job is to meet with Congress, we work with the committee's staffs that are responsible for appropriations. And we tell them about FDA, we answer their questions. We give them a feeling of what FDA is about, because again, it's not self-evident. One of the ways in which one of the things we've accomplished I mentioned the doubling of the appropriation of the taxpayer portion of the appropriation. Another thing we've done is we've changed the way Congress viewed views the agency or at least appropriators, when we started 15 years ago, we would just another responsibility in the Agriculture Appropriations Subcommittee. And, you know, that meant we were competing against farm programs, feeding programs, without any, you know, relationship to what the needs are those of those programs. What I think we've been able to do is where we were seen as not special. I think the committee has now realized that FDA is their special responsibility. And, you know, they may not give us the increases we want, but they do understand what FDA does, and they realize how disaster it would be disastrous it would be if they cut the agency.

So, the first part, as I said, is working with Congress. The agency has, you know, an expanding mission, it has growing responsibilities, we make sure They understand that those things don't happen without resources. And as Scott Gottlieb used to say, FDA can do more if it has more. And that sort of is an operative philosophy for us. The other thing we do is education and outreach to the media, to policymakers to the stakeholder holder community. That takes primarily two forms, one of which is we distribute analysis of FDA resource issues and funding issues. Every week in a publication called the Friday update goes to about 1000 people, reporters, stakeholders, every part of the and if people contact us, we're happy to put you on the mailing list for that. The other thing we've done is we've started to build a webinar series with senior FDA leadership. Yesterday, we had Dr. Califf on last year, we did 18 programs, with senior FDA leaders, including Dr. Califf appearing twice, every senator director appearing chief scientist, the head of Regulatory Affairs, and so on. And that program educates stakeholders about the agency. It educates the media about the agency. And then I get asked, because, okay, good lobbying and education. That's great, but what's the relationship, and to me, the relationship is evident, which is that the best argument for more funding of FDA is to understand how much it does. And that's the goal of the education program is to spread information on that, so that anybody who directly or indirectly is involved or hears things that are said in our forums, learns to understand how far FDA is affecting American society. It's obviously all of our food except meat. But it's the cosmetic products, probably every American is involved with an FDA product at the FDA regulate product at least a dozen times a day. And most of most people aren't counting most people aren't thinking about FDA. And it's our job to make them realize that that's how important FDA is to sterilize.

How many members does the Alliance currently have? Who are they?

We have 150 members of the alliance, we're on our way towards 200. Or we're doing some extra outreach this year. Plus, the webinars have made us better known and people stakeholders start to say, well, it Yeah, it matters to me, whether FDA has the resources to maintain the gold standard of regulation, it matters to me whether or not they're inspectors, it matters to me whether or not the agency can review products in a timely fashion when they come in, and etc. So that's the basis. I expect we're going to continue to grow as people recognize both us and the importance of our mission. And, you know, I whoever's listening to this, please contact us because every voice matters. There's no question about that. When we go down to the hill, and we're 200 members, it will be listened to better than when we have 150 members.

Then, of course, the part I mentioned, which is the fact that we're so diverse, reflecting FDA jurisdiction is always a good selling point. Any number of times that sat in a staffer’s office on Capitol Hill, and they're looking at our member list, and they say, you're right, Stephen, there couldn't possibly be anything else, that all the stakeholders all these interest groups agree upon. Other Then resources for FDA. And that's true. So, our members, our patient groups, as I've said, it's a variety that way. It's also in the other way in the sense that every part of FDA is part of the coalition. We have members who are interested primarily in food safety and nutrition. But also, we have members who are interested in vaccines, biologics, drugs, devices, over the counter products, dietary supplements, and not to be forgotten, because it is so important. FDA center bid for veterinary medicine is the primary regulator for animal feed, and animal medicines. And that's not just working animals, farm animals, it's also your pets. And at last count something in the neighborhood of north of 110 million households have a pet. So just right there, everybody has a stake in FDA, maintaining its gold standard and reassuring the American people, the products are safe.

What are the two guiding principles for the alliance?

When the Alliance came together, one of the important points was that we would stick to resources for the agency, because that's what every one of our disparate stakeholders agreed upon once if we were to get into policy matters, then there could be problems and the lesser effort would go towards resources. So that that was one of the principles was this is about appropriations. And in particular, FDA is the Agriculture Appropriations Bill and not in labor, HHS, not in the labor and Health and Human Services appropriations bill. So, it was one of the initial challenges when we came together was in fact, almost all the groups that were part of the Alliance had experienced lobbying labor, HHS, and who knew nothing about how the agriculture FDA Bill was different. So obviously, staying out staying focused on resources was one of the founding principles. Another was that budget authority appropriations, we just taxpayer money is vital to the American public and to the agency's public health mission. There are occasional weeds of interest in saying Well, shouldn't FDA be a user fee? Agency. And that comes from a very narrow view of what the agency does. User fees could not pay for nearly everything that FDA does.

So, while the alliance is focused on the taxpayer portion, which is the part that the Appropriations Committee focus on, we're supportive of all the elements of the $6.7 billion that FDA received in 2023. So public health mission, we've got to support the public health mission. It can't be done with user fees, I can assure you, user fees are necessary. They are important, essential, but they're things that can't do another principle was that the primary beneficiary of FTAs activities as the American public. That's the other more philosophical reason why user fees are appropriate, but shouldn't consume the agency, it's the publicans who benefits. And they need to continue to always have a say, in what the agency does. Another proudly principle was that the gold standard of the agency and its regulatory Do you know activities, science, public health, you know, always the gold standard, always insisting on evidence. And, you know, in safety, that that really applied to the entire agency and the food stakeholders had as much interested in that as medical product stakeholders. So that was important to our growth. 

Another is that a rising tide will lift all boats that every part of the FDA benefits from our advocacy. And we're going to stay that way. And the last, the last one, or the last two, actually, one is that FDA is the only federal regulatory agency that has the support of all of its safe stakeholders, including consumers and industry. And we wanted to preserve that that was very special. And then finally, you can't we were clear about a growing that FDA is growing responsibilities required a budget that grows. And that's been a mantra from the beginning. And we've been, you know, I want to be clear, we didn't do it, we can tribute it to a process that has led to adopt more than doubling of FTAs budget authority, taxpayer funding. And that comes from recognition, we certainly will take some credit, but it gets done because Congress understands it gets done because Congress cares. And those all come from stakeholders and consumers telling me that Congress that FDA matters. So, I think that's that segment, grayed out the goals of the Alliance.

The primary goal of the FDA, the Alliance for stronger FDA is increased resources. So, the agency can meet the needs of the American public. We do that a couple of different ways, one of which is directly with Congress. We're in constant touch with the hill and specifically appropriators. We have Hill days other outreach where our members communicate with Congress. And one of the best moments comes with a staffer realizes that industry group and consumer group are jointly asking for better funding from a regulatory agency. It kind of blows their mind, because that's not a meeting no matter how many years they've been on the hill. That's not a meeting they've ever been at. Because that's just not the outer world works. But it is part of what makes the FDA so special. In addition to the lobbying, and educating Congress, we also provide the stakeholder community with news and analysis about FDA resources. When we first started doing that people kind of steam How could you feel 52 issues a year talking about FDA resources? Well, when you move in the appropriations process, which Congress now is stretched out from a year to 15 to 18 months, and you look at the programming and how costs go up because of science becoming more complex, because the agency has more responsibilities. In fact, we produce those 45 or 46 issues a year, I need a few weeks off, but we have no problem filling it every week, week after week. That's how important FDA is. And that's how vast FDA is.

This kind of takes the form of the Alliance's Friday update, which goes out obviously on Fridays, and goes to about 1000 stakeholders and media now. If people get in touch with the Alliance, we'd be happy to add you to the list to receive that. We think people find it informative, and especially for those who want to understand how the funding comes about our coverage of how appropriate or see the agency is really quite thorough. Another vehicle about reach is that we run a speaker series a webinar series with senior FDA officials. Last year, we did 18 of them into including two appearances by Commissioner Kala and we're already off to a good start this year. We ended 2023 with a webinar with the new Deputy Commissioner for human foods, which is a new way of addressing food safety issues. And he was brand new, it was followed by Dr. Pete marks from the Center for Biologics, who talked about selling gene therapy. And FDA is position on this on the field, which is that there's a there's a chance it isn't going to work out and then FDA needs to work with industry to make sure those products meet the tests that are needed and come together and benefit patients.

So far this year, we've had a program on FTAs proposed reorganization, which will affect that 8,000 of FTAs 17,000 employees. Then yesterday we had Commissioner Caleb again with a fireside chat, upcoming programs we have our own policy legislative positions of FDA. We're going to be doing a program on siebers new super office which has all the selling gene products but also a lot of other cutting-edge technology under its jurisdiction. And then I'm hoping we're going to have a program on food chemicals Safety, which is one of the hot topics in the food safety area. So, all together, if you put the lobbying piece and the educational pieces together comes the question, you know, how do they relate? Why do you do both? And the answer is because the best argument for more FDA, more resources for FDA is, is to understand the incredible breadth of the agency. So, we educate on that, so that people say, oh, okay, that's why they need money because they do an X and Y and Z. Just a, for example, you know, 10 years ago, FDA wasn't worrying about medical software. Right now, that's important. devices come with software. AI is coming into the area, and FDA needs people who understand AI and can be involved in regulating it. Just as one example of how science becomes more complex, and the issues that FDA becomes part of become more difficult to just make a quick decision. So that's, that's really about our, our efforts. It's lobbying, it's the analysis, it's the education. And I think we do an important job. I mean, the agency has limits on what it can say it needs, which is normal, it's no different than any other federal agency. And so, one of our jobs through the webinar series, for instance, is to pick up topics that we know stakeholders want to hear about, that FDA is happy to have a forum to talk in. Its, you know, we invite them, we asked whether or not that would be something they supply a speaker for. And that's what makes the programs interesting. And that's what allows us to do 18 programs in one year.

What is the Alliance for stronger FDA hoping to accomplish within the next couple of years?

The environment for federal spending is going to get very tight, we're already seeing that. And so probably our most important goal over the next five years, is to make sure that there is widespread appreciation, particularly in Congress, about FDA special mission, the fact that it needs to happen, whether money is tight or not, and the job can't be done without resources. So that's, you know, we're coming into that environment and we're marshalling our forces to be more effective and making this into an issue that people recognize is important.

There are many federal agencies that if you said you're going to have 95% of the money you have to spend last year find 5% savings. They probably could do it. FDA is already starting and needs 10 to 20% more every year just because the science has become more complex. The areas it's being asked to regulate, become broader, and the reliance on safe food safe and effective medical products never slackens. Even while all those industries are growing. And so, one of the things I haven't mentioned that's important is also the appreciation that FDA is part of our commercial life in this country, that the industries that it supports through its regulation are ones that are creating jobs, that are an important part of the economy. And it, nothing, nothing knowing no one is there to do FDA is job if FDA doesn't have the funding that needs to do that job. And it's not a grant making agency, it's 80% of the FDS budget goes to people costs, and that salary and benefits and rent, training, travel, etc. But that's 80% of the agency's budget. So, if money doesn't come forward, it's not like oh, they can make fewer grants, because it's doesn't work that way.

So, I think more than anything over the next five years, we'd like to make the chorus the number of voices we have grown. And we'd like more appreciation to grow more appreciation of the fact that FDA is special, that if they're going to be across the board cuts, and they're saying, well, who is this problematic to the FDA comes to the top of the list and they say, well, we really can't do a cut at FDA. So that's the mission. It's to continue to expand the understanding and to translate that into congressional support that hopefully involves increased funding year over year.

Steven Grossman, Co-founder and executive director of the Alliance for a Stronger FDA, discusses the reason for founding the alliance, its members, goals, and the resources they offer. 

The Alliance for a Stronger FDA with Co-founder and Executive Director, Steven Grossman

Interview with Ashley Gaines of AstraZeneca

In an interview with Pharmaceutical Executive associate editor Don Tracy, Ashley Gaines, VP, head of breast cancer franchise, discusses newly approved Truqap.

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