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Rare Disease Leaders Call for Regulatory Clarity as FDA Balances Urgency with RigorApril 2, 2026 | 6 min read | Heather ...
07/04/2026

Rare Disease Leaders Call for Regulatory Clarity as FDA Balances Urgency with Rigor
April 2, 2026 | 6 min read | Heather McKenzie

With CBER director Vinay Prasad set to depart the agency at the end of the month, a coalition of patient groups and biotech executives penned a letter imploring the Trump administration to “restore regulatory clarity” for rare disease therapies. Experts last week also acknowledged the challenges faced by a more stringent FDA.
Rare disease drug developers rejoiced last year when the FDA issued multiple guidance documents aimed at accelerating therapies for these conditions to the market. In actual practice, however, there appears to be a disconnect between intent and regulatory decisions as the agency walks a precarious tightrope between scientific rigor and meeting unmet needs.
It's become more explicit that regulatory flexibility is appropriate, but not a waiver of rigor,” Rahul Gupta, President, GATC Health, said of the current FDA during a webinar hosted last week.
This balancing act has created strain between the FDA and patient groups and biotech companies working to advance rare-disease therapies. A coalition of advocates and executives this week penned a letter to the Trump administration imploring it “to restore regulatory clarity as it considers new leadership at the U.S. Food and Drug Administration’s Center for Biologics Evaluation and Research,” according to reporting by Reuters on Wednesday.
The letter, written by the Rare Disease Advocacy, Biotechnology, and Investor Coalition (RDBI), was sent to President Donald Trump, Health Secretary Robert F. Kennedy Jr., FDA Commissioner Marty Makary, and Medicare administrator Mehmet Oz.
The leadership transition referenced by the authors is presumably the one about to occur at the FDA’s Center for Biologics Evaluation and Research (CBER), which is largely responsible for regulating rare disease therapies. On March 6, news broke that Vinay Prasad—the controversial and embattled head of the division since last spring—will depart the agency at the end of April. Prasad took over as chief of biologics after the surprising departure of longtime, well-respected regulator Peter Marks, which sent the XBI biotech stock index plummeting to its lowest point in years. This is just one of many leaderships changes the agency has seen in the past year.
“I know that institutional transitions create policy ambiguity, even when the intent is aligned,” Gupta said during the webinar. “But the challenge right now is translating that FDA daily direction into actual, predictable, and operational guidance for sponsors.”
A More Stringent FDA
Over the past 11 months, Prasad has led a biologics division that has been seen by analysts and rare disease leaders as increasingly stringent and unpredictable, as guidance previously given by the agency is seemingly reversed, and therapies tested following that advice are rejected. Certainly, Prasad has been no friend of Capricor Therapeutics, Relumine, or Sarepta Therapeutics, among other rare disease biotech’s. The CBER director’s imminent departure “is a big win for biotech, especially for companies in the rare disease space,” Stifel analysts wrote in a note to investors on March 8.
In its letter, RDBI emphasized the lack of flexibility at CBER, and this consensus is reflected by the department’s recent approval record. In 2025, the division greenlit just five orphan drugs and issued four complete response letters (CRL), according to Stacey Frisk, executive director of the Rare Disease Company Coalition (RDCC), meaning that 44% of decisions made by CBER resulted in a rejection. That’s compared to the approximately 10% typically rejected in past years, Frisk said during the webinar. So far in 2026, the division has rejected two orphan products and approved one—Rocket Pharmaceuticals’ gene therapy Kresladi for leukocyte adhesion deficiency-I.
Meanwhile, CBER’s sister division, the Center for Drug Evaluation and Research (CDER) has greenlit four rare disease drugs, Frisk said, including Denali’s Avlayah for Hunter Syndrome.
“The landscape is a bit mixed,” she said.
A ‘Redefinition of Rigor’
Amid the inconsistency is another reality: novel therapies must be safe and effective. The question is how best to prove these qualities.
“The FDA is capable of doing two things: one, exercising regulatory flexibility; and two, complying with our obligation under the law to approve drugs based on ‘substantial evidence’ of effectiveness,” Makary noted in a March 25 press release announcing Avlayah’s approval.
Frisk lauded this statement, saying, “I think we’re seeing some encouraging signals.”
Gupta added that the FDA has been consistent in its assertion that companies can use external or natural history controls to support approval, “especially in rare diseases, but only when they’re fit for purpose and sufficiently reliable. The level of scrutiny around whether they truly meet that bar is what’s changed.”
For Matthew Winton, chief commercial and business officer at CervoMed, the FDA’s official pivot in February from a requirement of two pivotal trials to one for new drug applications represents a “redefinition of rigor,” putting more of an onus on the overall evidence package.
Instead of placing all the emphasis on one or two pivotal trials, regulators will be looking at “a package of data or totality of evidence to ensure that the drug is safe and efficacious for patients,” Winton said during the webinar. “I think it’s important to acknowledge that this is one of the things the FDA got right.”
Frisk noted that in this new paradigm, it is essential for rare-disease–focused companies to maintain early, continuous, and iterative dialogue with the FDA. “In rare diseases, we have different challenges,” she said. “We need to have the understanding that the expectation for a single perfect trial is going to be too high a bar for many rare diseases,” especially when considering novel endpoints and limited precedent and experience.
Capricor CEO Linda Marbán agreed, advocating for the use of external controls—comparing a cohort of patients receiving the investigational drug to a group of patients outside the trial. In guidance issued last year, the FDA itself encouraged the use of external controls in cell and gene therapy trials.
“Leadership is talking about history, real-world evidence, all of the abilities to use those data sets clearly to define the pathogenesis of a disease process as compared to some kind of a treatment paradigm,” Marbán said during the webinar. “That doesn’t seem to be translating down well into the review staff.”
Investors Crave Clarity
Rare disease drug development doesn’t fit the traditional biopharma investment model, representing another challenge for companies like Capricor, CervoMed, and the drug developers represented by the RDCC.
About two-thirds of biotech companies surveyed by RDBI said the uncertainty at the FDA had made it harder to raise capital over the past 12 months.
In this context as well, the single pivotal trial policy can be beneficial, according to Marbán.
“There are huge investment implications in one versus two pivotal trials,” she said. “I think that we, as a biotech community and world, have to acknowledge the fact that we are very dependent on investors.”
The old two pivotal trial format—which the panelists acknowledged was already being phased out, particularly in the rare disease space—required “a lot more investment and a lot more time that a company has to stay afloat,” Marbán continued.
The requirement for only one pivotal trial, she concluded, “really can give some great energy to the investment space if we have a total package that is based on rigor, but in addition to that, recognizing that there is an infusion of capital that’s necessary to move these programs forward.”
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While Johnson & Johnson retains the top revenue rank across the major pharma companies, Eli Lilly last year established ...
26/03/2026

While Johnson & Johnson retains the top revenue rank across the major pharma companies, Eli Lilly last year established itself as the clear leader in the obesity market, in the process capturing investors’ attention and enthusiasm.
2025 was Lilly’s year.
While the company wasn’t able to topple any record-holders—Merck’s Keytruda technically remains the top-selling drug, and Johnson & Johnson holds the overall sales crown—the giant from Indiana has been the year’s biggest buzz-maker.
In November, Lilly became the first pharma company to enter the trillion-dollar club, joining the tech-dominated pantheon of companies such as NVIDIA, Apple, Microsoft, and Alphabet with a market cap over $1 trillion. Pharma’s second-largest company is J&J, which, as of writing, has a market cap of $585 billion—far behind Lilly. J&J does more than just pharma, with a robust medtech section that contributes heavily to earnings.
While Lilly has consistently been a strong performer, its rise in 2025 was no doubt driven by strong ex*****on in the cardiometabolic space, particularly for the obesity and diabetes drug tirzepatide. In a recent interview, CEO David Ricks claimed that Lilly captures 70% to 75% of new patients starting their GLP-1 journeys— “almost three-to-one” versus its main competitor, Novo Nordisk.
That’s not to say other pharmas didn’t have a strong 2025. J&J and Pfizer stand out, for instance, for keeping earnings afloat despite persistent headwinds—be it an impending patent cliff, a continued post-pandemic slide, or the antagonistic rhetoric of a high-ranking health official. Others, still, have chosen to focus their efforts on building out their pipelines, paving the path for sustainable growth into the future.
Here, Arab Health World reviews the top five pharma companies in terms of revenue, looking at what’s behind their strong market performance in 2025 and how they plan to maintain their momentum moving forward.
J&J Keeps Crown Despite Stelara Loss of Exclusivity
2025 Revenue: $94.19 billion
YoY Growth: 6%
With money flowing in from both the pharma and MedTech industries, Johnson & Johnson is consistently among the top-earning drugmakers—and 2025 was no different.
With $94.19 billion in sales, J&J holds a comfortable lead over its Big Pharma peers (Roche is the closest challenger, more than $10 billion behind), despite the biosimilar erosion that immunology blockbuster Stelara endured in 2025. The product lost key market protections early last year and saw sales nosedive 41% from 2024.
J&J was well-prepared, however. In recent years, the pharma started cutting back on spending in some areas, such as infectious diseases and vaccines, and diverted these resources into immunology. The result is that newer products, including Tremfya and Simponi, have helped soften the blow of the Stelara cliff. Last year, these drugs surged nearly 40% and 22% year-on-year, respectively.
Meanwhile, J&J’s cancer division continues to be the cornerstone of the pharma’s business. In 2025, oncology accounted for more than a quarter of the company’s revenue, bringing in $25.4 billion—almost 21% growth from the previous year. The myeloma drug Darzalex was the pharma’s top-selling product last year, with $14.35 billion in earnings.
Medtech contributed $33.8 billion to J&J’s topline in 2025.
Roche Maintains Second-Place as Dealmaking Ramps Up
2025 Revenue: $79.48 billion
YoY Growth: 7%
Also racking up revenue from two industries—pharma and diagnostics—is Roche, which kept its second-place spot from the 2024 ranking. The group brought in CHF 61.516 billion ($79.48 billion)last year, representing 7% year-on-year growth.
Ocrevus, a monoclonal antibody indicated for multiple sclerosis, continued to be Roche’s top-selling product, making CHF 7.01 billion ($9 billion) in 2025, a 9% increase. The hemophilia A therapy Hemlibra likewise had a strong year, growing 11% to hit CHF 4.754 billion ($6.12 billion) in sales. Beyond these products, Roche attributed its revenue growth to the asthma drug Xolair, which surged 32% year-on-year to hit sales of CHF 3.075 billion ($4.78 billion).
Beyond its commercial performance, 2025 turned out to be a big dealmaking year for Roche. In September, the group acquired 89bio for $3.5 billion, obtaining its late-stage FGF21 analog pegozafermin for the treatment of metabolic dysfunction-associated steatohepatitis.
Roche in March 2025 also dropped $1.65 billion upfront—and promised up to $3.6 billion more in milestones—to partner with Zealand Pharma on its long-acting amylin analog petrelintide for obesity.
In October that year, during the pharma’s third-quarter earnings call, CEO Thomas Schinecker said that despite these hefty investments, Roche “is not done with BD.” The company has made good on that promise, with hundreds of millions poured into back-to-back cancer deals, as well as an RNA interference alliance.
To Third on Back of Obesity Rush
2025 Revenue: $65.179 billion
YoY Growth: 45%
With a 45% increase in revenue from the year prior, Eli Lilly is by far 2025’s fastest-growing pharma, jumping six spots to land in third place on this list. The Indianapolis powerhouse’s 2025 sales hit $65.18 billion.
Behind the company’s blockbuster year is the tirzepatide franchise. The drug, a dual agonist of the GLP-1 and GIP receptors, is marketed as Mounjaro for type 1 diabetes and Zepbound for weight loss. Mounjaro nearly doubled sales in 2025 to $22.97 billion, while Zepbound surged 175% year-on-year to make $13.54 billion.
Despite being a late entrant to the GLP-1 game—tirzepatide was first approved as Mounjaro in May 2022, while Novo Nordisk’s Ozempic was cleared in January 2020—Lilly has cleanly overtaken its competitor with a rapid ramp-up in sales that outpaced even Merck’s long-time cancer blockbuster Keytruda.
Also helping boost tirzepatide’s case are studies that show the drug to have better weight-loss and glucose control profiles than Novo’s semaglutide.
The future continues to look bright for Lilly’s business, as the lead over Novo extends in the obesity space. While the Danish competitor brought a weight-loss pill to the market first—the FDA signed off on an oral formulation of Wegovy late last year—Lilly’s own tablet, a GLP-1 analog called orforglipron, is close behind, with an FDA target action date of April 10.
Orforglipron also appears to have an efficacy edge over oral semaglutide, with the Phase 3 ACHIEVE-3 study demonstrating that Lilly’s pill elicited better sugar control and greater weight reduction.
Merck Slows Down, Focuses on Pipeline as Keytruda’s Cliff Approaches
2025 Revenue: $65.01 billion
YoY Growth: 1%
During Merck’s full-year 2025 earnings call, an analyst at TD Cowen pressed the pharma on its relatively slow growth last year: “Is this what we should expect from Merck going forward, a company that grows modestly in good times and is significantly pressured in less good times?”
Merck in 2025 was bogged down substantially by a slowdown in vaccine sales. Its HPV vaccine, Gardasil, made $5.23 billion globally last year—a 39% decrease from 2024. Pneumovax 23, the pneumococcal vaccine, cratered 37% year-on-year and brought in just $166 million in 2025.
Thankfully for Merck, the blockbuster PD-1 inhibitor Keytruda, which by now has become a cornerstone cancer therapy, continued its strong market performance, growing 7% to bring in $31.64 billion. Keytruda remains the industry’s top-selling drug, but it’s on a timer. Key protections for Keytruda are set to expire in 2028, after which sales are expected to erode as biosimilars enter the market.
Merck has been preparing for this, however, and last year secured an FDA approval for Keytruda Qlex, a subcutaneous formulation of the cancer therapy, which the pharma—and analysts—say will help cushion Keytruda’s fall off the patent cliff. The under-the-skin injection made $40 million worldwide last year in its initial months.
The approval of Keytruda Qlex is part of why CEO Robert Davis pushed back against the TD Cowen comment during the pharma’s investor call. “I’m not sure I agree with your characterization,” Davis told the analyst, insisting that 2025’s modest growth was being taken “out of context.”
In particular, Davis pointed to the pharma’s strong push to expand its pipeline—an effort that may not immediately reflect on the topline, but will nevertheless help keep earnings high past Keytruda’s lifespan. In July 2025, the company scooped up Verona Pharma for $10 billion, gaining the commercial drug Ohtuvayre for chronic obstructive pulmonary disease. Then, in November, Merck paid $9.2 billion to acquire Cidara Therapeutics for a late-stage antiviral drug.
These deals have helped Merck build what Davis said is “probably the broadest and widest pipeline we’ve had in years.”

Pfizer Pivots Focus to Obesity as Sales Continue Sliding
2025 Revenue: $62.579 billion
YoY Growth: -2%
Rounding out this list is Pfizer, which continues to struggle to regain its footing in a post-pandemic market. The pharma reported an overall 2% year-on-year sales slip to $62.6 billion, but taking its COVID-19 products—the antiviral Paxlovid and the vaccine Comirnaty—out of the equation reveals 6% operational growth.
Comirnaty nevertheless remains one of Pfizer’s best-selling products, making $4.37 billion worldwide. The blood thinner Eliquis is the company’s top seller, with $7.96 billion for 2025, followed by the Prevnar family of conjugate vaccines with $6.49 billion in sales.
Notably, with vaccines accounting for much of its earnings, Pfizer has been particularly exposed to policy headwinds in the U.S., driven by continued critical rhetoric and controversial vaccine policies from Health Secretary Robert F. Kennedy, Jr. So much so, in fact, that Pfizer CEO Albert Bourla felt the need to speak up about it at the World Economic Forum in January.
To return to growth, Pfizer is leaning heavily into the already-lucrative but increasingly competitive obesity market. The pharma previously tried to advance its own GLP-1 drug danuglipron, but was forced to pull the plug amid safety issues. Late last year, Pfizer won over Novo in a public bidding war for Metsera, ultimately paying $9.8 billion to acquire the biotech.
The pharma has since put more money into its weight-loss pipeline, including a potential $1.9 billion deal with China’s YaoPharma in December 2025 for an investigational GLP-1 drug, as well as a $495 million partnership with Sciwind Biosciences in February to license a GLP-1 injection called ecnoglutide, which is approved in China for diabetes.

Gene Therapies for Hearing Loss Strike an Encouraging Note in Embattled ModalityMarch 23, 2026 | 7-minute read | Eli Lil...
25/03/2026

Gene Therapies for Hearing Loss Strike an Encouraging Note in Embattled Modality
March 23, 2026 | 7-minute read |

Eli Lilly and Regeneron are leading the push to treat congenital deafness with gene therapies, seeking a piece of a potential billion-dollar market and banking on local delivery and the small amount of drug required to overcome key safety concerns.
The last year has been challenging for the gene therapy space.
A series of deaths—headlined by the high-profile case of Sarepta’s Elevidys—has cast doubts about safety, while rejections, delays, and study holds have dampened the economic case for developing gene therapies. But there is one disease area where the modality is thriving: hearing loss.
“The stage is set to make an impact in the auditory area with gene therapies over the next decade,” Jonathon Whitton, vice president and global program head for Genetic Medicines at Regeneron, said. Regeneron is competing with Eli Lilly to bring the first gene therapy for deafness to the market.
The company’s DB-OTO, which aims to restore the expression of otoferlin—a protein encoded by the OTOF gene that is critical to the hearing process—in patients with congenital deafness. DB-OTO—elicited “clinically meaningful” improvements in hearing within weeks of dosing in a Phase 1/2 trial in October 2025.
Meanwhile, Lilly’s AK-OTOF, a dual adeno-associated viral vector-delivered gene therapy, restored the hearing of a child in January 2024.
“Millions of individuals worldwide have disabling hearing loss because one of their genes generates an incorrect or incomplete version of a protein [otoferlin] the ear requires for hearing,” a Lilly spokesperson said. AK-OTOF, they continued, delivers a functional copy of the otoferlin gene to elicit “durable expression of functional otoferlin protein to the inner hair cells of the cochlea.”
There is currently no treatment for the approximately 1 in 500 infants in the U.S. who are born with some form of deafness each year, Whitton said. Genetic anomalies account for more than half of these cases, he continued, which can be diagnosed with a saliva sample and genetic testing.
This means that the potential market “is huge,” Zheng-Yi Chen, associate scientist at the Eaton-Peabody Laboratories at Massachusetts Eye and Ear, “which is attractive for the pharma/biotech industry.”
Indeed, the global market for hereditary deafness is expected to exceed $1 billion by 2033, up from $610 million this year, according to a recent report by Coherent Market Insights.
Lilly and Regeneron are both well-positioned to take advantage. AK-OTOF’s Phase 1/2 study is expected to wrap up in October 2028, while a regulatory package for DB-OTO was filed late last year, with a decision expected in the first half of this year.
Success in the hearing loss space could have positive implications across gene therapy, experts agree.
A Safety Audible
In theory, “applying gene therapy to treat hearing loss or deafness makes sense” for several reasons, not least of which is the monogenic nature of the disease, according to Graig Suvannavejh, managing director of Equity Research, Biopharmaceuticals and Biotechnology at Mizuho Securities.
This is true for congenital forms of hearing loss, at least. “It’s important to keep in mind that for the other 50% of patients with hearing loss, their condition came from acquired causes,” Suvannavejh clarified in an email. “So, a gene therapy won’t help them.”
Aside from the relatively simple genetics of the disease, however, the cleaner safety profile of ear-targeted gene therapies is another key advantage, Suvannavejh said.
Chen agreed, explaining that the heaviest toxicity concerns with gene therapy stem from adeno-associated virus (AAV) vectors, which are used to package the genetic payloads and protect them from temperature and chemical assaults when inside the body.
Last summer’s Elevidys saga shone a harsh light on the use of AAVs, highlighting their propensity to accumulate in the liver and damage the organ. AAVs are also highly immunogenic, meaning they trigger a response from the immune system, which can further damage the liver or even spiral into a more systemic reaction like a cytokine storm.
“In most cases where death has been reported, AAV was administered systematically in large quantities, which resulted in toxicity,” Chen said. “For diseases where local delivery is feasible, such as the eye and the inner ear, the risk is greatly reduced.”
Hearing loss is a perfect test case for this, Chen and Suvannavejh agreed. Not only is the delivery of gene therapies for deafness very localized, but it can also be done via well-established surgery techniques, further minimizing injuries that can come from risky procedures, Suvannavejh said.
In addition, as deafness is not a fatal condition, patients enrolled in gene therapy trials for hearing loss are “relatively healthy,” the Mizuho analyst continued, meaning they are easier to treat and suffer from fewer comorbidities and complications.
Listening to the Market
Another key allure of the hearing loss space is its attractive market proposition.
In its report, Coherent Market Insights recognized that precision therapies have the “potential to revolutionize treatment” for hereditary deafness. This will drive demand for targeted treatments, the market intelligence firm said, much like the ones Lilly and Regeneron are developing.
Both companies have recognized the potential returns and have invested accordingly. Lilly, for instance, bought into the field in October 2022, when it acquired Akouos in a $487 million cash transaction, gaining access to AK-OTOF. The pharma has continued to pump money into its hearing loss precision portfolio, betting up to $1.3 billion in May 2025 for access to Rznomics’ RNA editors. Earlier this year, Lilly again put more than $1 billion on the line to advance site-specific recombinases with Seamless Therapeutics.
Regeneron gained ownership of DB-OTO when it swallowed partner Decibel Therapeutics for $109 million in August 2023.
Gene therapies for hearing loss are less costly to bring to market than those for other indications, according to Suvannavejh. “The amount of drug needed for a hearing loss gene therapy is very small,” he explained, “So the cost of goods is low—probably much lower relative to other gene therapies that need to be delivered systemically.”
Regeneron’s Whitton agreed, noting that the requirement of just a small amount of the therapeutic material makes things a bit easier for drugmakers. “Drug development is a tough industry,” he said. The ability to keep doses low, along with local delivery, “helps to address many of the challenges that systemic gene therapies have faced.”
An Ear to the Future
More broadly, the current hearing loss push could have far-reaching implications for the gene therapy space as a whole, Whitton said.
“There is something powerful about the broader momentum that progress creates,” he said. “A meaningful result in one area, even a rare one, can show the field what is possible and give others the confidence to take similar chances.”
One example of this is the creation of dual-AAV gene therapies, Chen said, which were “first applied successfully to genetic hearing loss.” Because this platform uses two vectors, it is able to introduce large genes that wouldn’t have otherwise fit into a single AAV package, according to a 2019 study published in PNAS, which used this technique to deliver two halves of the otoferlin gene into mice. The genetic payload was successfully reconnected in the animals’ inner ear, reversing deafness.
Another mouse study published in Clinical and Translational Medicine in January validated the dual-AAV approach in hearing loss, showing that the platform can deliver the entire length of the STRC gene—also commonly mutated in congenital forms of deafness—into the animals, likewise restoring hearing.
While the technology is still in its infancy, the dual-AAV approach has “opened a new avenue for efficacious treatments,” Chen said.
Italian biotech AAVantgarde is trying to bring this platform into the clinic. Its lead program targets Usher syndrome, a rare genetic disorder that causes partial or total hearing loss and progressive blindness. The company completed enrollment in a Phase 1/2 study in this indication in January.
Aside from dual AAVs, Chen pointed to other novel modalities, such as antisense oligonucleotides, gene editors, and RNA therapies, which he believes will also be leveraged for hearing loss in the coming years.
Suvannavejh is similarly excited about the field’s future, though more cautiously so.
“I am not particularly sure about potential read-throughs to gene therapies for other conditions,” he said, again referencing the localized delivery of hearing loss therapies—a key distinction he insisted makes them “rather unique” from systemic genetic medicines.
“That said,” Suvannavejh continued, “I think any positive advancement in gene therapy for hearing loss . . . will likely only stimulate more interest by both industry and the investment community in gene therapies for hearing loss, as well as in the gene therapy space overall.”

Validated Mechanisms, Strong Data Beat Hype in Longevity InvestingMarch 23, 2026 | 3 min read | Jennifer C. Smith-Parker...
24/03/2026

Validated Mechanisms, Strong Data Beat Hype in Longevity Investing
March 23, 2026 | 3 min read | Jennifer C. Smith-Parker,
Longevity is a long-standing buzzword in life sciences, but it now has staying power. The smart trajectory is to stop chasing aging as an abstract target and concentrate on specific mechanisms that can clearly advance specific, age-related diseases, according to two investors in a discussion.
Roughly a decade ago, longevity burst into headlines as the next big thing in biotech. But the hype fizzled not because of a lack of robust science, said Sergey Jakimov, managing partner at LongeVC, a venture capital firm focused on biotech and longevity investing. Rather, start-ups failed due to an unfocused strategy that viewed slowing aging as a vague, monolithic goal rather than a focus on aging-related issues to be measured, regulated, and covered by payers.
Ideally, longevity can be considered the investigation and understanding of underlying mechanisms across several age-related disease categories, to impact a wider patient population and prolong lifespan, Jakimov said.
Regulatory and Payer Paradigms
Since aging is not classified by regulatory authorities as a disease, companies need to think in standard clinical development terms of age-related conditions, said Artem Trotsyuk, U.S. operating partner at LongeVC. Longevity players need to “speak the FDA language” as any other biotech and develop data-driven trials with measurable endpoints and post-approval requirements, Jakimov and Trotsyuk agreed.
Hence, a realistic path for a longevity start-up is to pursue a specific disease target and validate that mechanism rooted in aging biology, Jakimov said. In practice, that research must be proven like traditional drug targets, he added. Variability in aging reinforces the need for developers to focus on concrete indications with mechanistic clarity, Trotsyuk added.
Once a drug is approved, payers expect real-world outcomes data, and longevity companies must build their evidence strategies early to meet this, Trotsyuk said.
GLP-1s: Longevity Poster Child?
GLP-1 agonists might be considered the first successful longevity product, Jakimov said. The drug class started with a narrow indication- diabetes- and then expanded to obesity, once not considered a stand-alone indication, Trotsyuk said. GLP-1s now have potential broader systemic health perks, such as cardiovascular, kidney, and Alzheimer’s disease benefits, they agreed.
GLP-1 programs focused on specific mechanisms and receptor pathways early, whereas longevity companies cannot target aging as it doesn’t exist as a mechanism, Trotsyuk said.
Longevity players should follow the GLP-1 playbook, Jakimov said. First, they should demonstrate solid data in a specific condition, then claim wider health benefits and expand said indication once initial sales and channels are in place. To make a commercial dent, longevity developers need to understand large pharma behavior in terms of large-scale distribution, market access, and control of patient and provider channels, Jakimov said.
Pitch Do’s and Don’ts
Longevity start-ups need to have a clear story in their pitch decks, rather than chase buzzword trends, Trotsyuk said. In that vein, LongeVC avoids investment in TikTok-style health trends and nutraceuticals that lack data, even if they are safe or popular, Jakimov said.
The firm’s broad remit covers therapeutic and nontherapeutic interventions, tools, diagnostics and measurements that capture what it means to be healthy and age well.
Firstly, LongeVC considers the potential asset’s health space, such as cardiovascular disease, and the desired indication to pursue. It then benchmarks the company’s approach against others in the same space it has already seen. A close eye is paid to whether so-called novel technology is just a repackaged older approach and the extent of development progress made in a certain period, Trotsyuk said.
In due diligence stage, consideration is given to data appearing in paper, internal reports and in detailed mechanism explanations. A feasibility demonstration is crucial: while there is more flexibility at the concept stage, there is less tolerance for thin data by Series A/B.
Red flags include platforms that generate hits, targets, or molecules without a clear drug product, Trotsyuk said. Company teams also must be realistic about the timelines, regulatory hurdles, and business development involved, Jakimov said. Teams that want to avoid engaging industry now and assume deals will come quickly later, despite deal cycles typically taking a year or more, arouse skepticism, he added.
Although healthspan endpoints may eventually be recognized by regulators and payers, the path to real longevity currently runs straight through classic biotech discipline.

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