Makary's New FDA: Expose Corruption, Buy These Biotechs Now
For decades FDA decisions have been scrutinized. Many labeled it as a corrupt institution where favor was curried for lucrative positions in big pharma after FDA service. Critics have also pointed out the financial incentives held by advisory committee members were highly conflicted in the sense that grants or consulting fees were tied to the very drugs they were evaluating. The latest FDA commissioner Marty Makary wants to change the narrative and prove to the world that the FDA is capable of efficiently approving drugs while keeping the status of an unbiased watchdog over safety. His immediate agenda is hyperfocused on getting drugs approved and he has telegraphed his targets. The biotechs mentioned at the end of this article represent asymmetrical risk/reward.
Breaking the Public Trust
During the pandemic the FDA moved at unprecedented speed utilizing Emergency Use Authorizations (EUA’s) to lower the approval bar in order to get vaccines approved. From a legal standpoint, an EUA only requires that the product “might work” and that its known benefits outweigh the risks. At the time, there was a massive wave of “groupthink." Leaders decided that vaccines were the only way to deal with COVID-19 despite the availability of low cost alternatives like Hydroxychloroquine, Ivermectin, Tollovid, and Quercetin/Zinc.
Because the government was so focused on vaccines, they exploited legal loopholes to protect the big pharma players. This meant vaccine makers like Pfizer and Merck couldn’t be sued if anything went wrong. The result was a massive real-world clinical trial without the back up of enough safety data. The FDA’s main job is to look out for people but they decided to favor corporate profits and legal protection during COVID-19. This was a massive breach of fiduciary oversight that led to an unacceptably high rate of vaccine injury that broke the public’s trust in the agency.
Fixing the Mistakes of the Past
Before Makary started his push for “Radical Transparency" the FDA was mired in black box decision making whereby all the inputs go into a regulatory black box and then the FDA issues a binary verdict without any accountability. The pandemic era FDA was a failure of both science and transparency. The Makary FDA is positioning itself more as a regulatory watchdog ensuring safety which is actually its congressional mandate.
Public Disclosure of FDA Letters: The FDA approval process has been binary for decades. Drug sponsors would get either an approval with a specific label or they get a rejection letter called a Complete Response Letter (CRL). In the past the CRL was a private matter between the drug company and the FDA which details the safety failures or efficacy shortfalls. This allowed both the drug companies and the short activists like STAT and Hindenburg Research to spin the story to their advantage. New FDA rules require the public release of a CRL in real-time.
FDA Advisory Committee Overhaul: Advisory committees were assembled without conflict checking the members and whether they had direct or indirect financial interests in the drugs they were evaluating. The integrity of advisory boards was often questioned. The new FDA rules prevent employees of drug companies from serving on advisory committees. The goal is to restore public trust in the process.
Phasing Out Animal Testing: Many drugs at the beginning stages were required to do expensive animal testing that didn't really transfer to humans. Millions of dollars would be spent on these tests only to see them fail in humans raising the overall cost of drug development. In place of these animal studies the FDA has shifted toward New Approach Methodologies (NAMs) that use either AI-modeling or “organ-on-a-chip” These changes enable startups to bypass the time intensive animal testing gauntlet and use their resources more efficiently. This move levels the playing field for innovative biotechs and chips away at the moat that Big Pharma had at stifling competition.
Hunting for Companies w/ Priority Review Vouchers
Dr Makary is on the hunt for drugs that fit the following conditions:
PTSD Blindness
Mental Illness Deafness
Cancer
In his address he talked about a treatment for genetic hearing loss under the National Priority Voucher program. Regeneron Pharmaceuticals (NASDAQ: REGN) makes a gene therapy called Otarmeni. Decibel Therapeutics was a public company before being acquired by REGN in 2023 for $213 million. This biologic is designed to treat kids with congenital deafness. The drug received FDA approval and REGN got a Priority Review Voucher worth around $200 million or more based on recent sales. Big pharmas are not the way to play the new FDA but elements of future targets are on display for all to see.
The Formula
Priority Review Pathway, Orphan Drug Status, Pediatric, Gene Therapy, Focus Diseases
The FDA is looking for drugs with a priority review pathway and this classification of drugs typically translates into an orphan drug. Orphan drugs are drugs with a treatment population under 200,000 patients per year. These rare diseases are typically treated with monoclonal antibodies delivering gene therapy.
OS Therapies (NYSE: OSTX)
OS Therapies is aggressively advancing its lead immunotherapy candidate, OST-HER2, for the treatment of recurrent pulmonary metastatic osteosarcoma. This is a pediatric disease which makes them eligible to earn a PRV under the Rare Pediatric Disease Designation (RPDD) Program. The company is currently in the midst of a rolling Biologics License Application (BLA) submission with the FDA, targeting an accelerated approval pathway. They also started prepping their regulatory filings in Europe, the U.K., and Australia. They have a platform technology based on bioengineered Listeria monocytogenes bacteria. They have enough cash to last them into 2027. The company had excellent Phase 2 data that showed 75% progression free survival versus the historical 40% at 3 years. The results were statistically significant and with the new FDA perspective on approvals, it's a compelling play for investors focused on rare, high-unmet-need pediatric and adult oncology indications.
Capricor Therapeutics (NASDAQ: CAPR)
Capricor is fast approaching its PDUFA date on August 22, 2026, regarding its Biologics License Application for deramiocel in Duchenne muscular dystrophy. There are rumblings of an early approval. There is an opening at the FDA for a new head of CBER and Houman Hemmati is one of the leading candidates. Look at his comments from January basically pleading to get approval of deramiocel.
While this news has already bumped the stock, the CBER position hasn’t been filled. For those concerned about valuation, there's way more in the tank and here’s why. Capricor has one of the most powerful platform technologies in the field of exosome therapy. Deramiocel is a cellular therapy but it uses the lungs as a bioreactor to create the exosomes that help treat the heart in Duchenne Muscular Dystrophy (DMD). Exosomes can be used for many forms of treatment and as a drug delivery system.
An approval might also send related names in the exosome space higher. So keep an eye on Aegle Therapeutics (NASDAQ: AGLE) which has MSC‑derived exosomes for regenerative and dermatologic indications. Coya Therapeutics (NASDAQ: COYA) – has an exosome‑based platform targeting neurodegenerative and inflammatory diseases. Nurexone Biologics Inc. (OTC: NRXBF) developed a targeted exosomal therapy that can help regenerate spinal cord injury. Zeo Scientifix Inc. (OTC: ZEOX) developed a line of exosome based supplements and a biologic called Zofin to treat ARDS, TBI, Arthritis, and COPD. LAMY Inc (OTC: LMMY) is developing a novel exosomal gene therapy to treat Glioblastoma.
ImmunityBio (NASDAQ: IBRX)
The face of ImmunityBio is led by Dr. Pat Soon-Shiong who is a high profile surgeon, oncologist, and business man. He is a passionate cancer advocate looking for common sense solutions to treat cancer. Their leading drug candidate Anktiva is an IL-15 receptor agonist that works by attracting Natural Killer (NK) cells to treat cancer patients. Anktiva is currently approved in the U.S., UK, EU, and Saudi Arabia for BCG‑unresponsive non‑muscle invasive bladder cancer. In the USA they are working on metastatic Non-Small-Cell Lung Cancer (NSCLC). In a podcast he implied that Ankitiva could work in all cancers and forgot to use the disclaimer “in theory.” The FDA sent out a warning letter in March 2026 regarding Dr Pat's podcast “Is the FDA BLOCKING Life Saving Cancer Treatments?” After the warning, the company walked back what it said, but if the FDA is all about transparency then the results will ultimately speak for themselves. IBRX is one of the first NK therapies targeting cancer and “in theory” it could be used on all types of cancers and undergo a massive label expansion. This is a massive platform technology that could be tested on a number of different cancer indications.
Revolution Medicines (NASDAQ: RVMD)
Revolution Medicines is a company that is working on a treatment for RAS addicted cancer. RAS cancers are cancers that have this mutation that makes them highly dependent upon the RAS protein for growth. Block this protein and the tumor halts its growth or dies off. They have a Phase 3 trial in metastatic Pancreatic ductal adenocarcinoma (PDAC) and a phase 1/2 in Non-Small-Cell Lung Cancer (NSCLC). There is also an early trial in Colorectal cancer (CRC). Positive Phase 3 results for its lead candidate, daraxonrasib (RMC-6236) showed the drug had an overall survival benefit (OS) of 13.7 months vs 6.7 months. These results put them in contention for an acquisition, but they have a market cap of around $30 billion and have been the topic of merger discussions in that same price range so it's hard to think of what premium needs to get paid to do a deal. It was recently reported that suitors include Merck (NYSE: MRK) and AbbVie (NASDAQ: ABBV).
Erasca (NASDAQ: ERAS)
Erasca continues to advance its RAS-targeting franchise, with recent data showing encouraging early clinical activity for its molecule ERAS-0015 across multiple tumor types. With both ERAS-0015 and ERAS-4001 moving through clinical trials, the company is executing on its 2026–2027 milestones, maintaining a steady pace as it works to validate its potential as a provider of best-in-class therapies for solid tumors.
Neurogene Inc. (NASDAQ: NGNE)
Neurogene is focused on developing gene therapies for rare, devastating neurological disorders like Rett syndrome. They use a needle to deliver a viral vector directly into the fluid filled space of the brain. This delivers the replacement gene so the cells start producing the full length gene enabling the cells to produce the missing protein needed to reverse the disease. The company is in a phase 1/2 clinical trial and close to completing enrollment. Interim data showed 100% functional improvement and 88% achieved an improved CGI-1 score and the safety was “well tolerated.” They need a 35% response rate to meet their endpoint at 12 months. If interim results continue this is almost a no-brainer. The next major update comes during the summer. Rett Syndrome is one of the larger rare diseases affecting about 6-9k patients in the USA. It checks the boxes for the Makary FDA. It's a gene therapy, a brain disease, NGNE has the Rare Pediatric Disease (RPD) designation which can get them PRV. The therapy appears to be well tolerated and there’s no real treatment so this looks like an ideal candidate for FDA approval.
MeiraGTx (NASDAQ: MGTX)
MeiraGTx is advancing its gene therapy pipeline with a strong emphasis on regenerative medicine, including positive data releases in early 2026.The FDA as it develops solutions for complex, rare conditions.ting neurological disorders, highlighted by its lead program for Rett syndrome. The company distinguishes itself through its proprietary transgene regulation platform and in-house manufacturing capabilities, leveraging clear regulatory designations to advance its pipeline within the specialized genetic medicine landscape.
Profiting Off of Transparency
Lets face it the opportunities in biotech were only available to those on the inside of politics. It wasn’t a level playing field because the truth could be hidden depending on the political agenda. There’s a new sheriff in town who wants to do the right thing. Dr Makray seems to have leveled the playing field for investors where hard work and due diligence can pay off but don’t be naive. He wants wins, which translates into drug approvals. He’s telegraphing what he's looking at next. He also wants the USA to win back its credibility as the world’s leading regulator. So pay attention to the man, following his breadcrumbs could lead you to some monster wins in your portfolio. Pediatric - Gene Therapy - Rare Disease - Voucher Program are all breadcrumbs leading to one name OSTX one of the best bang for the buck plays in biotech.