Moderna, Inc.

iStock, CoreDesignKEY Policy initiatives have come fast and furious at the FDA this year. While guidances on rare diseases and vaccines have consumed most of the ink, policy shifts aimed at improving FDA efficiencies and reshoring U.S. manufacturing also got some attention. Here, BioSpace rounds up more than a dozen initiatives relevant to the biopharma industry. From vaccines to rare diseases and the phasing out of animal testing requirements, it was a busy year for biopharma on the policy front as Health Secretary Robert F. Kennedy Jr. and his team at HHS made their mark on the FDA, CDC and National Institutes of Health. Many of the FDA’s 2025 guidances have focused on accelerating the path of cell and gene therapies to the market—particularly those intended for rare and ultrarare diseases. In a year where controversy is almost expected with every news announcement, the need for more and more-effective therapies for rare conditions may be the one area on which Americans of every political stripe can agree. Analysts and other FDA watchers who spoke with BioSpace see the momentum generated in this area in 2025 continuing into the new year. “I do think that, given what we’re seeing from FDA, rare orphan diseases as an area” of great interest for biopharma and investors will persist into 2026, Graig Suvannavejh, managing director of Equity Research at Mizuho Securities, told BioSpace . Married with reports that the FDA will soon begin requiring only one pivotal trial as opposed to the standard two, “this would make it seem ripe for rare orphan [diseases],” he added. Another key area of focus for the HHS under Kennedy—with his long history of antivaccine activism—has been in the vaccines space. On April 30, the Health department announced it would require all new vaccines be tested in placebo-controlled trials before they are approved, a “radical departure” from past practices, an HHS spokesperson told The Washington Post at the time. Earlier this month, Kennedy’s reformulated CDC Advisory Committee on Immunization Practices made another radical departure from the norm, voting to delay the hepatitis B vaccine from birth to two months of age for some infants. “Obviously [Kennedy is] driving a lot of this, from vaccine policy to staffing and everything like that,” Chad Landmon, Hatch-Waxman & Biologics chair at Polsinelli Law Firm, told BioSpace , “so I think it’ll be interesting to see how that continues, whether the same people continue in those roles.” BioSpace has compiled a review of our coverage of this year’s key policy initiatives. We will continue to update this tracker as we move into 2026 and see what comes next. _______________________________________________________________________ Randomized Trials To Be Required for CAR Ts December 8 Randomized, controlled clinical trials will be the FDA’s preferred approach to assessing CAR T cell therapies, Center for Biologics Evaluation and Research (CBER) director Vinay Prasad and three co-authors wrote in a Perspective piece in the Journal of the American Medical Association . Single-arm trials with no controls —the type on which the approvals for the seven currently marketed products were based—will no longer be sufficient. The FDA will also look at the therapy’s benefit in terms of patient survival or on time to the development of a particular event when considering approval, according to the authors. Prasad and colleagues noted in the piece that while the FDA is adjusting its processes to “exercise regulatory flexibilities, when necessary,” it also needs to “maintain the high evidentiary standards for approval.” FDA CAR-T Developers Will Need Randomized Trials as FDA Eyes Tighter Approval Requirements For traditional approval, CAR T therapies will need to establish superiority over current standard treatments, including already-approved CAR T products. December 9, 2025 · 2 min read · Tristan Manalac _______________________________________________________________________ FDA To Reduce Pivotal Trial Requirement From Two to One December 4 In possibly its biggest overarching pivot of 2025, the FDA is planning to require only one pivotal trial, as opposed to two, for companies seeking approval of a new drug. While not yet an official policy—the change was communicated by FDA Commissioner Marty Makary to STAT News in an interview—he told the publication it will be finalized in the next three to six months. “You can achieve the same statistical power with one trial as you would with two,” Makary told STAT . While analysts and other insiders positioned the proposed change as a major coup—biopharma consultant David Alderman in a LinkedIn post called it the “biggest jailbreak in biotech history”—other experts told STAT they were confused because the FDA already allows flexibility in situations where running two trials would be impractical or infeasible. _______________________________________________________________________ FDA Unveils Plausible Mechanism Pathway for Rare Diseases November 13 Makary and Prasad introduced the plausible mechanism approval pathway for rare diseases. Writing in The New England Journal of Medicine , Makary and Prasad said the new pathway would expedite treatments “for products where a randomized trial is not feasible.” In an interview with Fox News in April, Makary introduced the concept of a “conditional approval” pathway for treatments for rare diseases with extremely small patient populations. When the detailed pathway was announced seven months later, Makary and Prasad referenced Baby KJ , a patient with the ultrarare disease CPS1 deficiency whose condition was treated with a custom gene editing therapy. To qualify for the new plausible mechanism pathway, therapies must meet several conditions similar to KJ’s, including targeting known biological causes for a specific disease, and sponsors must produce post-marketing data to show real-world efficacy. FDA Makary’s ‘Conditional Approval’ Pathway for Rare Diseases Poses More Questions Than Answers While sparking excitement among biopharma companies focused on rare and ultrarare indications, experts say FDA Commissioner Marty Makary’s proposal is light on details and raises potential concerns about safety, access and liability. May 19, 2025 · 10 min read · Heather McKenzie _______________________________________________________________________ Guidance To Prevent Refusal-to-File (RTF) Letters October 23 The FDA released draft guidance to help biopharma companies understand what the agency considers to be a complete regulatory submission, and conversely, what will result in a refusal-to-file (RTF) letter. The guidance also explains the difference between filing issues that would result in an RTF and review issues that would result in a rejection. Recommendations include that a drugmaker not submit “materially incomplete or inadequately organized” applications, and that applications using a “single adequate and well-controlled clinical investigation” as the sole basis for approval will be RTF’d if the FDA and the applicant had previously agreed on the need for more than one such study. FDA FDA Provides Guidance To Prevent Returned Applications, Aid Patient-Focused Drug Development In three draft guidance documents, the FDA has laid out pointers to help companies craft better drug development programs and drug applications. October 24, 2025 · 2 min read · Tristan Manalac _______________________________________________________________________ Abbreviated NDAs Aim To Speed U.S. Generics October 3 Just over a week after its guidances for speeding the development of CGT therapies, the FDA on Oct. 3 introduced a new program for accelerating the generic drug industry in the U.S., both on the R&D and manufacturing sides. The agency’s new program includes a quicker review of abbreviated new drug applications —specific regulatory filings for generics—though the FDA did not say by how much it would accelerate review processes. Reviews for generic medicines generally take about 10 months. To qualify for that speedier review, companies must conduct their bioequivalence studies in the U.S. and “exclusively” source their active pharmaceutical ingredients from within the country as well. The finished dosage form must also be made locally. 2025 data showed that most generics are manufactured China and India, representing 22% and 44% of the global market, respectively. Manufacturing FDA Continues US Manufacturing Drive With Domestic Generics Pilot This latest FDA program aims to provide speedier reviews for generic drugmakers who produce their products in the U.S. October 6, 2025 · 2 min read · Tristan Manalac _______________________________________________________________________ FDA Seeks To Streamline Cell, Gene and Regenerative Therapies September 24 In a set of three separate draft recommendations, the FDA published new thinking on how to accelerate development of cell and gene therapies and regenerative medicines. These guidances covered a range of topics, including leveraging “innovative” study designs that use patients’ status prior to a trial as their own control. Other recommendations included using historical or real-world data as controls, allowing mid-trial modifications as studies generate data and deploying new mathematical frameworks to model progressions of a disease to chart the therapy’s efficacy. The agency also addressed diseases with small study populations, stating that early-stage trials could be enough to establish a product’s efficacy given “appropriate quality controls,” as well as post-marketing requirements for CGT products approved under accelerated pathways. FDA FDA Details Thinking for Streamlining Development of Cell, Gene, Regenerative Therapies Three draft recommendation documents published on Wednesday are intended to guide drug sponsors and accelerate the development of cell and gene therapies. September 25, 2025 · 2 min read · Tristan Manalac _______________________________________________________________________ FDA Cracks Down on “Deceptive” Drug Advertising September 9 The FDA on Sept. 9 announced it was sending approximately 100 cease-and-desist letters to pharmaceutical companies with what it called “deceptive advertisements.” The agency would also initiate rulemaking to close the “adequate provision” loophole, created in 1997, that has allowed pharma companies to advertise their drugs without fully disclosing risks, “fueling inappropriate drug use and eroding public trust,” according to the FDA’s statement. However, according to experts involved with writing the 1997 regulation who spoke with BioSpace , it was not a loophole. Instead, it was a deliberate and carefully crafted law that struck a happy medium between the demands for marketing new drugs and informing patients. FDA In New DTC Ad Crackdown, FDA Trying To Close a Loophole That Isn’t While the FDA is trumpeting this new initiative as “sweeping reforms” to the way drug companies can advertise, experts say the regulator is going after a problem that doesn’t exist. September 17, 2025 · 7 min read · Annalee Armstrong _______________________________________________________________________ FDA Unveils Rare Disease Evidence Principles September 4 The FDA’s Center for Biologics Evaluation and Research (CBER) and Center for Drug Evaluation and Research (CDER) on Sept. 4 jointly unveiled a new framework intended to streamline the approval of therapies for ultrarare diseases. The Rare Disease Evidence Principles (RDEP) will allow companies developing certain therapies to approval under an investigational new drug application using only a single-arm trial. This will apply to sponsors developing drugs for diseases that affect a “very small, rare disease population . . . generally less than 1,000 persons in the United States,” according to the FDA’s announcement . Qualifying conditions must also be linked to a known genetic defect and characterized by progressive functional deterioration leading to disability or death “in a relatively short period of time.” The targeted diseases should also lack “adequate” alternative therapies. Rare diseases Rare Disease Experts See Futility in FDA’s Proposed Framework The FDA’s proposed Rare Disease Evidence Principles review process is a starting point for getting rare disease therapies across the finish line, but industry leaders say there are more concrete steps the regulator could take to help patients. September 22, 2025 · 5 min read · Dan Samorodnitsky Rare diseases Saol To Pitch Ultrarare Disease Drug as “Poster Child” for RDEP Program Saol Therapeutics received a complete response letter for its pyruvate dehydrogenase complex deficiency treatment a week after the FDA unveiled its Rare Disease Evidence Principles program. On Dec. 18, in a Type A meeting, the biotech will attempt to convince the agency that its drug fits perfectly into the framework. December 9, 2025 · 2 min read · Heather McKenzie _______________________________________________________________________ FDA Pledges to Release CRLs in Real Time September 4 The FDA promised that, going forward, it would release new complete response letters (CRLs) to the public “ promptly after they are issued to sponsors .” In July, in a bid for “ radical transparency , ” the FDA released a bundle of more than 200 CRLs from a variety of drug and biological products applications submitted to the agency between 2020 and 2024. Big Pharmas like Eli Lilly and Regeneron were represented, as well as smaller biotechs like Sarepta. At the time, the FDA said the CRLs released were an “initial batch” without elaborating on its plans. Alongside the FDA’s September announcement, it released a second cache of heavily redacted CRLs, for products including Lykos Therapeutics’ MDMA-based therapy for posttraumatic stress disorder and Stealth BioTherapeutics’ elamipretide for Barth syndrome, approved later that month on a second try. As of December, the archive of CRLs has grown to 392. Complete response letters FDA Publishes Cache of Complete Response Letters in Bid for ‘Radical Transparency’ The trove of more than 200 letters is part of a pledge of transparency from the agency, with the intention to increase public insight into the reasons new drug and biologics applications got rejected. July 10, 2025 · 2 min read · Dan Samorodnitsky FDA Wall Street the Main Beneficiary of FDA’s Real-Time CRL Disclosures By publishing complete response letters as soon as they are issued to drug sponsors, the FDA is expanding transparency in a way that, while positioned as a public health measure, also grants investors greater visibility into regulatory decisions. Experts question whether this is the agency’s proper remit. September 29, 2025 · 6 min read · Ben Hargreaves _______________________________________________________________________ FDA Introduces PreCheck Program To Boost US Drug Manufacturing August 7 The FDA unveiled the PreCheck program with the intention of supporting biopharma companies as they build new facilities in the U.S. The PreCheck program provides manufacturers with more frequent FDA communication at critical development stages, including facility design, construction, and pre-production. A second Application Submission Phase centers around streamlining development of the Chemistry, Manufacturing, and Controls section of the application through pre-application meetings and early feedback. The program follows an executive order issued by President Donald Trump in May instructing the FDA to cut down on the 5–10 years it currently takes to build pharmaceutical manufacturing plants in the U.S. The EO also set the table for the FDA to implement surprise inspections on foreign plants, as opposed to the planned visits that are the current standard operating practice. “A scheduled visit is no inspection,” Makary said from the White House on May 5. The order will also see inspectors spend less time in the countries where the inspections take place, allowing them to “get in and out,” conducting more inspections with the same amount of resources, Makary said. The action is just one of many Trump has undertaken this year aimed at onshoring pharmaceutical manufacturing. Manufacturing Generic Drug and API Manufacturers Call for Onshoring Incentives Having seen Congress spend money to onshore semiconductor production, pharma groups are pushing for similar incentives for domestic drug manufacturing. November 4, 2025 · 4 min read · Nick Paul Taylor Manufacturing Pharmas Hope FDA PreCheck Can Help Avoid Facility-Related Drug Rejections Executives from Eli Lilly, Merck and other companies foresee the FDA’s new onshoring proposal being anything from a bureaucratic waste of time to a transformative program that will eliminate inspection-related complete response letters. October 28, 2025 · 4 min read · Nick Paul Taylor _______________________________________________________________________ Commissioner’s National Priority Voucher Program Introduced June 17 The FDA’s Commissioner’s National Priority Voucher (CNPV) program is intended to shorten review timelines for certain drugs from 10–12 months to 1–2 months. The initiative streamlines reviews by assembling a team-based review in a one-day “tumor board style” meeting, rather than shuttling applications sequentially through offices of the agency. For drugs to be awarded a CNPV, they must be “aligned with U.S. national priorities,” according to the FDA. Those priorities could include addressing a national health crisis, delivering “more innovative cures for the American people,” addressing an unmet public need or increasing domestic drug manufacturing. The FDA announced an initial batch of nine recipients in October, with an additional six awarded in November. A month later, the agency awarded its first CNPV approval of a generic antibiotic produced by USAntibiotic. Regulatory FDA Hands Out First Commissioner’s Priority Vouchers to Regeneron, Sanofi, Disc, Others The Commissioner’s National Priority Voucher awards companies that align with specific national priorities—such as boosting domestic manufacturing and lowering drug prices—with faster reviews and more frequent interactions with the FDA. October 17, 2025 · 2 min read · Tristan Manalac Regulatory Pressure On, Purpose Questioned as Second Round of FDA Priority Review Vouchers Revealed Priority voucher awardees and regulators could feel pressure to “meet the moment” as FDA watchers question the intent and feasibility of the Commissioner’s National Priority Voucher program. November 17, 2025 · 6 min read · Heather McKenzie Regulatory Wegovy, Casgevy Among Latest FDA Priority Review Voucher Recipients The FDA awards a second round of Commissioner’s National Priority vouchers to six larger biopharma companies. And this time, with the exception of Eli Lilly’s orforglipron, the vouchers are for drugs that are already on the market. November 6, 2025 · 3 min read · Dan Samorodnitsky _______________________________________________________________________ FDA Allows Drug Imports From Canada May 21 Drug pricing has been a top agenda item for the first year of President Trump’s second term. In May, Trump signed an executive order instructing U.S. health agencies to attempt to lower drug prices through whatever means are available to them. The FDA responded to that memo with a guidance that allowed states and Indigenous tribes to import prescription drugs from Canada, with the aim of lowering drug prices “without imposing additional risk to public health and safety.” In its announcement, the FDA said it anticipated meeting with states that expressed interest in the import program in the fall, though no such meetings have been announced. The Canadian government, for its part, made it clear that it would not export drugs to the U.S. “if that sale would cause or worsen a drug shortage in Canada,” according to a statement provided to BioSpace at the time. Drug pricing FDA Advances Drug Importation Plan as Canada Moves To Protect Its Supply Canada’s health agency says it has been “taking all necessary action safeguard the drug supply and ensure Canadians have access to the prescription drugs they need.” May 22, 2025 · 2 min read · Annalee Armstrong _______________________________________________________________________ HHS Requires Placebo-Controlled Trials for New Vaccines April 30 The Department of Health and Human Services announced it would require all new vaccines be tested in placebo-controlled trials before they are approved. While vaccines for new pathogens are already tested against placebo, new vaccines for other diseases are evaluated against existing interventions, largely for ethical reasons. The new policy “forces researchers to unethically withhold proven protection from some patients, all under the guise of ‘scientific rigor,’” Larry Bucshon, a cardiothoracic surgeon and former Congressman, wrote in an opinion article for STAT News . An HHS spokesperson told The Washington Post that the flu vaccine would be exempt from the new requirement as it “has been tried and tested for more than 80 years.” In a note to investors on May 1, analysts at Leerink Partners called the new policy “negative news for vaccine manufacturers,” including Merck, BioNTech and Moderna. Policy HHS To Require Placebo-Controlled Trials for All New Vaccines in ‘Radical Departure’ From Past The new policy, revealed by the Department of Health and Human Services on Wednesday, would make vaccines “less available and less affordable,” according to vaccine expert Paul Offit. It could also further complicate the picture for companies like Novavax. May 1, 2025 · 3 min read · Heather McKenzie _______________________________________________________________________ Restricting Pharma Representation on Adcomms April 17 One of FDA Commissioner Marty Makary’s earliest moves was to limit the ability of pharmaceutical company employees to serve on agency advisory committees . Even on committees where they are statutorily allowed to serve, the FDA said it would “prioritize and elevate” others on adcomms, particularly patients and caregivers. The agency noted that the change in policy did not preclude employees of regulated companies from attending or presenting to adcomms, and that exceptions could be made to allow their service on committees if they had unique expertise on a topic. This change was an early example of Makary’s efforts to “boost public trust” in the FDA, as the agency’s announcement put it. FDA FDA Moves To Restrict Industry Influence on Advisory Committees Industry representatives will still be allowed at these meetings, but they will no longer have a spot on the advisory committee. April 18, 2025 · 2 min read · Tristan Manalac _______________________________________________________________________ FDA To Begin Phasing Out Animal Testing April 10 In a move the FDA and U.S. government had been building toward for years, the agency announced plans to phase out its animal testing requirements for monoclonal antibodies and other drugs. The FDA plans to replace these animal models with “more effective, human-relevant methods,” such as AI-based computational models of toxicity, organ-on-a-chip systems and organoids. By leveraging nonanimal models, in addition to real-world human data, “We can get safer treatments to patients faster and more reliably, while also reducing R&D costs and drug prices,” Makary said in a prepared statement. “It is a win-win for public health and ethics.” Clinical Research FDA, NIH Accelerate Shift Away From Animal Research as Experts Warn of Limitations The FDA and NIH recently announced plans to phase out animal testing requirements for some therapies. While organoid and AI providers celebrate, scientists warn that questions over safety, applicability and implementation remain. May 19, 2025 · 6 min read · Natalia Mesa FDA New Approach Methodologies: Redefining Animal Testing Alternatives In this episode of Denatured, Jennifer C. Smith-Parker speaks to Stacey Adam, PhD, Vice President of Science Partnerships at the Foundation for the National Institutes of Health and Patrick Smith, Senior Vice President, Translational Science at Certara, to discuss the latest regulatory news and the future for new approach methologies (NAMs) development. December 4, 2025 · 1 min read · BioSpace Insights

iStock, quantic69 Pfizer and Metsera, Sarepta and uniQure made the list with dramatic tales. The other two spots went to the regulatory challenges facing biopharma under the new administration, especially in the vaccines sector. A constant refrain over the past year in the BioSpace newsroom chat has been, “Will this news cycle ever slow down?” Well, we sit here in late December with the answer: No. December has been anything but slow, with market-moving, hotly anticipated data still coming in, regulatory and personnel drama at the FDA and CDC, and much more keeping this little newsroom busy. The truth is, we thrive on this stuff and while we may throw up our hands from our keyboards on some mornings—and Friday afternoons—and ask the news gods, Why? , we secretly love every second of it. So going into the holiday break, the BioSpace news team took a look back at the stories we thought were the most important from 2025. These sagas intersected all of our beats, from business to clinical development to the regulatory environment. FDA FDA Policy Tracker: 2025 Was a Year of Change Policy initiatives have come fast and furious at the FDA this year. While guidances on rare diseases and vaccines have consumed most of the ink, policy shifts aimed at improving FDA efficiencies and reshoring U.S. manufacturing also got some attention. Here, BioSpace rounds up more than a dozen initiatives relevant to the biopharma industry. December 22, 2025 · 13 min read · Heather McKenzie Read More Pfizer-Metsera-Novo Bidding War Fit for Reality TV Where does one even begin with the saga that was the Metsera acquisition? Or as BMO Capital Markerts analyst Evan David Seigerman dubbed it: The Real Housewives of Biopharma . In September, Pfizer offered a cool $4.9 billion upfront plus a contingent value right to buy obesity hotshot biotech Metsera. The company debuted in 2024 to much fanfare, then met the hype with multiple clinical readouts that quickly put it as the front of the pack of up-and-comers in the ballooning weight loss space. It conducted one of the most successful IPOs of 2025 in January. The Pfizer buyout seemed on its way to closing. In regulatory filings about the deal, Metsera noted that there had been other parties interested, including one—later revealed to be Novo Nordisk—that was likely to have regulatory challenges in closing. Then Novo dropped a bomb . On October 30, the Danish pharma, desperate to replenish a flagging obesity pipeline, submitted an unsolicited bid to usurp Pfizer and buy Metsera for about $8.5 billion. A normally restrained Pfizer seemed livid, calling the offer “reckless and unprecedented.” The New York pharma quickly filed litigation against Novo, Metsera and the biotech’s shareholders. A bidding war began, with both parties upping the ante all the way to $10 billion. Novo’s CEO even called out Pfizer in an appearance at the White House: “Our message to Pfizer is that if they would like to buy the company, then put your hand in the pocket and bid higher,” Maziar Mike Doustdar said from behind a lecturn in the Oval Office. Days later, Pfizer finally secured its prize. The company matched Novo’s $10 billion bid but offered something Novo could not: certainty. Pfizer had already secured the Federal Trade Commission’s blessing. Novo was likely to take as long as two years to finalize the deal, using a framework that had already drawn antitrust concerns from the regulator. Metsera shareholders approved the deal and it closed in November, thereby capping off the most exciting biopharma M&A story in years. Obesity Is Metsera Worth the Fuss? Pfizer and Novo Nordisk seem to want Metsera bad. Analysts are wondering, though: Is the obesity biotech really worth this much effort? November 5, 2025 · 4 min read · Annalee Armstrong Sarepta’s Gene Therapy Platform Causes Deaths and Drama This summer, Sarepta Therapeutics found itself in hot water with investors and the FDA after failing to disclose the death of a patient taking one of its investigational gene therapies. News of the death broke via media reports the day after the company announced a black box warning for its approved Duchenne muscular dystrophy (DMD) gene therapy Elevidys, following two earlier deaths tied to the treatment. The company also stated that it was pivoting away from the vector technology used for both gene therapies and for which Sarepta had received the first-ever FDA platform designation. Sarepta CEO Doug Ingram later told investors that the company didn’t disclose the death in this corporate update “because it was neither material nor central to the topics at hand.” Sarepta had previously reported the death to the FDA, but the agency later claimed “it was lost in the bureaucracy” and that the agency had only found out about the third death with the rest of the world. Communication mishaps aside, the FDA reacted to news of the third death by pausing clinical trials for the investigational therapy, revoking Sarepta’s platform designation and asking the company to discontinue shipments of Elevidys. To that final request—which was a request and not a demand because Elevidys has full market approval—Sarepta initially said no . But after just a few days, the company relented, and paused Elevidys shipments for all U.S. patients for 10 days . In November, the FDA made official the black box warning for Elevidys and limited its use to ambulatory patients. Sarepta’s future now hangs in the balance. After starting the year with a share price of $124, the company’s stock hit its lowest point in nearly 10 years and continues to hover around just $20. Editorial Lack of Transparency Tarnishes Sarepta’s Sheen as Patient Deaths Trigger FDA Battle Sarepta Therapeutics’ stock has dropped precipitously as questions swirl around the safety of its gene therapies. Meanwhile, the Duchenne patient community fears losing access to Elevidys while the regulator considers more drastic action. July 22, 2025 · 6 min read · Jef Akst UniQure Brings Hope in Huntington’s but Highlights Regulatory Woes While the Alzheimer’s and amyotrophic lateral sclerosis spaces have seen incremental breakthroughs over the past five years, patient with Huntington’s disease have continued to wander in a desert of hopelessness . Until this September. That’s when uniQure reported three-year data from a pivotal trial of its gene therapy AMT-130 showing 75% slowing of the devastating neurodegenerative disease. While small—the Phase I/II study consisted of just 29 patients—experts hailed the moment as “historic” and BBC News declared that Huntington’s had been successfully treated for the first time. Still, Ignacio Munoz-Sanjuan, CEO and chairman at Rumi Scientific and president of the Huntington’s-focused non-profit Factor-H, cautioned against communicating the results in a way that conveyed unrealistic expectations at this stage of development. The space had been here before. In an interview with BioSpace , Munoz-Sanjuan recalled a similar reaction to Roche’s tominersen when it was shown to lower mutant huntingtin (mHTT) levels in the cerebrospinal fluid in a Phase I trial. Tominersen would later fail in Phase III. Still, uniQure’s trial was considered pivotal and the company communicated plans in a Sept. 24 press release to FDA approval of AMT-130 in the first quarter of 2026, with a potential launch later next year. Five weeks later, the FDA said not so fast. On Nov. 3, following a pre-BLA meeting, uniQure revealed that the agency “no longer agrees” that the Phase I/II data for AMT-130 will be “adequate to provide the primary evidence in support of a BLA submission.” This, despite the company having aligned with the agency on the protocols and statistical analyses used. This news ultimately placed uniQure at the heart of another key story of 2025—apparent FDA 180s faced by several biotechs, including Replimune, Biohaven and Capricor Therapeutics. Since uniQure’s latest announcement, more than 10,000 Huntington’s patients, families and supporters have signed two petitions calling for the FDA to reconsider its latest guidance. “For families who have long awaited new treatment options, the FDA’s actions feel like a serious setback and have raised urgent concerns about transparency, consistency, and trust in the regulatory process,” Christina DeGryse, a Huntington’s disease advocate, wrote in an email to BioSpace on Nov. 16. FDA FDA Reversals Send UniQure, Biohaven, Capricor, More Into a ‘Tailspin’ Since July, several biotechs have been forced to pivot as previous agreements with the FDA around evidence required for approval were reversed, a phenomenon that, according to experts, could portend a more restrictive regulator. November 24, 2025 · 7 min read · Heather McKenzie FDA Turmoil Creates Uncertain Regulatory Environment What is FDA policy in 2025? It’s the operational question of the year for biopharma. Proposals for regulatory changes have come via unusual channels—on social media, in media interviews and in editorials published by medical journals—often with little detail about how the new policies will be implemented. In BioSpace ’s Policy Tracker, we attempted to round up all the changes the industry has faced this year, but due to the uncertainty of these communications, it was often difficult to decide whether or not to include an initiative introduced by Commissioner Marty Makary, Center for Biologics Evaluation and Research (CBER) head Vinay Prasad or other key regulators. Promo: Policy Tracker (Notably, the CDC has faced similar challenges with communication, with former CDC Chief Medical Officer Debra Houry telling Senators that she learned of changes to the CDC’s COVID-19 vaccine guidance from an X social media post—messaging she said she could not use to implement new public health guidance.) Setting aside the unusual modes of communication, policies implemented by the FDA in 2025 have caught their share of controversy; from the requirement announced in April that all new vaccines be tested in placebo-controlled trials to the introduction of the Commissioner’s National Priority Voucher program in June—a program whose intent and feasibility has been questioned by experts . One area where industry responded with some positivity was in the cell and gene therapy space—particularly around those therapies being developed for rare and ultrarare diseases—where myriad initiatives and guidances focused on accelerating their path to the market. The FDA’s Rare Disease Evidence Principles framework, introduced in September, is intended to streamline the approval process for therapies targeting diseases affecting less than 1,000 people in the U.S., while a trio of guidances published later that month elucidated the FDA’s thinking on how to speed cell and gene therapies specifically. But many questions remain about the implementation of these and other policy changes, especially in light of unprecedented churn at the agency, which has experienced a near-complete turnover of senior leadership since Donald Trump was elected U.S. president. BioSpace learned in the spring after the forced resignation of CBER director Peter Marks that more than half of FDA leaders had left the agency since the presidential election. By December, after the sudden retirement of newly appointed CDER Director Richard Pazdur, that number was up to almost 90%. Only three names on the organizational chart from a year ago remain today. Going into 2026, industry is calling for consistency and stability at the U.S. drug regulator. Vaccines in the Cross Hairs of Health Secretary Robert F. Kennedy Jr. Observers feared that the Trump administration, along with new Health secretary and frequent vaccine opponent Robert F. Kennedy Jr., would make sweeping changes to federal vaccine policy. The administration validated that concern almost immediately. In a post on X in June, Kennedy announced that he was removing the CDC’s COVID-19 vaccination recommendation for healthy children and pregnant women. A few weeks later, he removed all 17 members of the CDC’s influential Advisory Committee on Immunization Practices, replacing them with new members more aligned with his antivaccine views. Over the summer, Kennedy also pulled back funding from vaccine research, to the tune of $500 million for mRNA-based projects. That funding would’ve gone to vaccine makers like AstraZeneca, Moderna, Pfizer and Sanofi but was pulled because “the data show these vaccines fail to protect effectively against upper respiratory infections like COVID and flu,” Kennedy said in a statement from HHS, without providing evidence. The drama continued in August, as newly confirmed CDC head Susan Monarez was fired after refusing to pre-approve ACIP vaccine recommendations, she alleged. The following month, ACIP recommended splitting up the stalwart MMRV—measles, mumps, rubella and varicella (chicken pox)—shot into two separate MMR and V doses. And in its most recent meeting in December, the committee recommended delaying the hepatitis B vaccine birth dose—in use for 30 years with no prior issues—to two months post birth for babies whose mothers test negative for the virus, a move that came amid multiple condemnations from former CDC and FDA chiefs on the CDC’s policy changes. The CDC formally adopted that recommendation shortly afterward. To cap the year off, the FDA’s Vinay Prasad wrote, in a leaked internal memo, that COVID-19 vaccines had been linked to at least 10 pediatric deaths, without offering evidence. The FDA promised that that data would come soon. Soon after, news broke that the FDA planned to put a black box safety warning on COVID-19 vaccines—though in the meantime, the CDC published data showing that COVID-19 vaccines are effective at protecting children against disease. Eleven days after the FDA promised to release data relevant to Prasad’s claim, a report broke that internal research at the agency suggested the number of deaths linked to COVID-19 vaccines was between zero and seven, and that none of the seven potential deaths were conclusively tied to vaccination. The following day, Makary contradicted the reports of the FDA putting a black box warning on COVID-19 vaccines. Shares of Moderna and fellow vaccine biotech BioNTech both dropped about 20% this year, while four pharma giants— GSK , Pfizer, Sanofi and Merck—all reported declining vaccine sales due to dropping immunization rates in the U.S. Vaccines Pesky ‘Macro Factors’—aka RFK Jr.—Come for Vaccine Pharmas’ Wallets Following restricted vaccine approvals and changes to CDC immunization schedules, Merck, Pfizer, GSK and Sanofi are all suffering revenue hits to their vaccine programs. November 7, 2025 · 4 min read · Annalee Armstrong

和铂医药是一家典型的 “平台型Biotech” （生物科技公司），其投资逻辑与信达生物等已有产品的公司完全不同。它的核心价值不在于当前营收，而在于其 全球领先的技术平台的价值发现和变现潜力 。这是一笔 高风险、高潜在回报 的投资，更像是对其技术平台的“期权”。 一、 核心优势与投资亮点（Bull Case - 看多的理由） 全球顶尖的全人源转基因小鼠平台（H2L2平台） 这是和铂医药最核心的资产。该平台能够高效生成全人源单克隆抗体，相比传统方法，产生的抗体免疫原性更低、成药性更高。 价值证明 ：该平台已经成功催生了 特那西普（针对干眼症） 等进入临床III期的产品，并吸引了大量全球顶级药企的合作。平台的实力已得到验证。 强大的对外合作（BD）能力：真正的“卖水人” 和铂的战略非常清晰：利用自身平台，为全球大药企“淘金”提供“铲子”。通过 授权合作（License-out） 获取首付款、里程碑付款和未来销售分成。 成功案例 ： 将一款 CLDN18.2xCD3双特异性抗体 （HBM7022）授权给 莫德纳（Moderna） ，总交易额超 10亿美元 。 与多家巨头（如辉瑞、阿斯利康、礼来、百时美施贵宝等）达成数十项合作。 意义 ：这些合作不仅带来了宝贵的现金流，减轻了融资压力，更是对其技术平台的 顶级国际背书 ，证明了其平台的商业价值。 富有潜力的自研管线（尽管已大幅精简） 公司战略转型后，聚焦于最有潜力的核心资产。 特那西普（HBM9036） ：治疗中重度干眼症，已处于全球多中心临床III期。这是一个巨大的市场，如果成功，商业价值巨大。但该领域竞争激烈，结果是“赢家通吃”。 HBM7008 ：一款针对B7H4x4-1BB的双特异性抗体，具有“条件性激活”的独特机制，有望在提高疗效的同时降低毒性，目前已有早期临床数据读出，前景被市场看好。 财务安全垫相对充足 通过多次成功的BD授权交易，公司获得了可观的 预付款 ，使其现金流相较于许多同类Biotech更为健康， “烧钱”速度得到控制 ，降低了近期内的融资破产风险。 二、 潜在风险与挑战（Bear Case - 看空的理由） 尚无商业化产品，缺乏自身造血能力 公司至今 没有任何产品上市销售 ，所有收入均来自合作授权款，没有稳定的销售收入来源。本质上仍是一家“烧钱”的研发公司。 核心自研产品面临高风险和高竞争 特那西普（干眼症产品） ：III期临床数据存在不确定性。干眼症赛道竞争极其激烈，已有多个产品上市（如环孢素Xidra、Eysuvis等），失败风险高。这是公司最大的“赌博点”之一。 其他自研管线都处于较早阶段，距离上市变现遥遥无期，且同样面临临床失败的风险。 股价历史表现疲软，流动性较差 作为港股18A公司，和铂医药股价经历了长期下跌，从上市初的高点回落甚多。市值较小， 日均成交额和换手率较低 ，流动性不足。这意味着大资金进出困难，股价容易大起大落。 估值高度依赖“故事”和BD进展 由于没有盈利和销售收入，其估值完全建立在 市场对其平台未来能签下多少BD协议 以及 自研管线临床数据是否成功 的预期上。一旦BD合作放缓或出现临床失败，股价容易遭受重创。 对合作伙伴的依赖性 虽然BD合作是优点，但也意味着部分价值的实现依赖于合作伙伴的研发和商业化能力。如果合作伙伴决策迟缓或投入不足，也会影响里程碑款的收取。 总结与展望 和铂医药不是一家传统的Biotech，而是一家“技术平台型”公司。 它的投资逻辑非常清晰： 看多逻辑 ：投资它，就是投资其 H2L2等技术平台能持续不断地产生优质资产 ，并吸引全球大药企前来合作。你赌的是它成为“Biotech界的台积电”或“抗体领域的卖水人”，通过持续的BD交易实现价值。下一个与Moderna级别的合作公告可能就是股价的催化剂。 看空逻辑 ：它的自研管线可能失败，BD合作可能放缓，现金流可能再次紧张。在没有自身造血能力的情况下，长期来看仍可能需要通过稀释股权的再融资来维持运营，从而摊薄现有股东权益。 投资者类型匹配： 适合 ：对生物科技行业有 深度理解 、能承受 极高风险 、追求高潜在回报的 “风险投资型”投资者 。这类投资者看重平台价值和BD潜力，而非短期利润。 不适合 ： 保守型投资者 、寻求稳定股息和短期回报的投资者、以及对生物医药研发高风险特性不了解的投资者。 你需要密切关注的关键点： BD合作新闻 ：是否有新的授权交易公布，交易金额大小（首付款尤其重要）。 临床数据读出 ：特别是特那西普的III期临床数据结果，这是决定其自研管线价值的生死线。 财务报告中的现金储备 ：查看期末现金及等价物余额，计算其“现金流跑道”（还能支撑运营多久）。 大股东和机构动向 ：是否有知名机构增持或减持。 总而言之，和铂医药是一只 “高赔率但低确定性” 的股票。它拥有顶尖的技术，商业模式也得到了初步验证，但最终能否为股东创造巨大价值，仍取决于其能否持续地将技术优势转化为商业成果。在考虑投资前，你必须做好本金可能部分甚至全部损失的心理准备。