CSL Ltd.

iStock, NicoElNino The FDA’s refusal to review Moderna’s mRNA-based flu vaccine is part of a larger communications crisis unfolding at the agency over the past nine months that has also ensnarled Sarepta, Capricor, uniQure and many more. What do 2025’s Sarepta saga and 2026’s “Modernagate” have in common? While each case is unique, they both exposed a glaring trend: a lack of effective communication from the FDA—a subject that also featured prominently in a panel discussion I hosted at Phacilitate’s Advanced Therapies Week in San Diego this week focused on Geopolitics, policy, and the new global landscape for Advanced Therapies . On Tuesday, Moderna received a refusal-to-file (RTF) letter from the FDA for its mRNA flu vaccine mRNA-1010. While most news coverage has focused on the administration’s stringent policies and negative rhetoric regarding vaccines—particularly those that are mRNA-based—this incident mirrors a larger theme unfolding at the FDA over the past nine months: the agency reversing its position on evidence required for product approval. “It should not be controversial to conduct a comprehensive review of a flu vaccine submission that uses an FDA-approved vaccine as a comparator in a study that was discussed and agreed on with CBER prior to starting,” Moderna CEO Stéphane Bancel said in a statement on Tuesday in response to the RTF. At this, executives from several biotechs—including Capricor Therapeutics, uniQure and Biohaven—were likely nodding their heads in empathy. Reversals of guidance from the FDA have heavily impacted the approval timelines of Capricor’s Duchenne muscular dystrophy (DMD) cell therapy deramiocel, Biohaven’s troriluzole for spinocerebellar ataxia and uniQure’s Huntington’s disease gene therapy AMT-130. 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 For Sarepta’s part, the lack of communication followed the deaths of two teenage patients who were treated with its approved DMD gene therapy Elevidys. On July 18, 2025, Sarepta acknowledged receipt of an “informal request” from the FDA to voluntarily halt shipment of the product for all DMD patients. However, the company said it “first heard of this potential request earlier in the day at the same time the public and our patient communities did, through media reports.” Different situations; same game of broken regulatory telephone. Inconsistent Guidance As is the case with Moderna, much of the regulatory confusion appears to emanate from the Center of Biologics Evaluation and Research (CBER), which oversees the review of biologics, including most cell and gene therapies and vaccines, and is largely focused on trial design. “I can say definitely that’s not what the field in rare disease needs,” Samar Mohanty, president & chief scientific officer at Nanoscope Therapeutics, said during Tuesday’s panel discussion about the lack of regulatory clarity. Mohanty noted that at CBER specifically, the FDA is “directed towards evidence-based science. . . . They want randomized control trials.” But this is not always feasible, he added, hence previous guidance that biomarkers are sufficient in certain rare diseases. Such previous guidance was allegedly given to uniQure regarding the registrational trial for AMT-130. In November 2025, the company received FDA feedback indicating that data from the Phase 1/2 studies of the candidate in comparison to an external control—what’s known as a single-arm study—may not be adequate to support a biologics license application (BLA) submission. CEO Matt Kapusta expressed surprise with the feedback, which he said in a statement at the time was “a drastic change from the guidance the FDA provided in November 2024 that data from the ongoing Phase 1/2 studies, compared to a natural history external control, may serve as the primary basis for a BLA submission under the accelerated approval pathway.” Huntington’s disease FDA Does ‘About-Face’ on UniQure’s Huntington’s Gene Therapy A month after sparking optimism for patients with Huntington’s disease with highly positive data for AMT-130, uniQure revealed Monday that the FDA may be changing its tune on the evidence required for an approval application, a change of direction Stifel called “very challenging” for uniQure. November 3, 2025 · 3 min read · Heather McKenzie Not to mention from CBER’s most recent guidance. In September last year, CBER issued a series of guidance documents intended to speed the approval of cell and gene therapies, given the “urgent need for safe and effective” medicines to address severe conditions affecting small populations. One guidance document endorses single-arm trials using participants as their own control. Atara Biotherapeutics’ T cell immunotherapy Ebvallo, being proposed to treat a serious post-transplant complication related to the Epstein-Barr virus, was also turned back by the FDA due to a single-arm study. The agency’s “new position is contrary to the FDA’s prior guidance to Atara, the FDA’s alignment with Atara on the clinical trial data set, and the acceptance of the trial design as a single arm study as relevant for this patient population at BLA submission,” the company said in a Jan. 12 press release announcing the complete response letter. Some of this could be due to a lack of clarity regarding how to implement the guidance. “One of the challenges we see is that as regulations, as guidances have come out, they aren’t easily implementable,” Roberta Duncan, former chief scientific officer at Arcturus Therapeutics, said during the Advanced Therapies Week panel. The FDA’s plausible mechanism pathway for ultra-rare diseases, outlined in an article published in The New England Journal of Medicine in November 2025, “is a great first start, but there is an inability to actually implement that that needs to be figured out,” she said. ‘A Good Partner’ For the most part, however, the panelists agreed that the FDA is trying. Mohanty defended the agency’s more cautious approach. He pointed to the recent clinical hold placed by the FDA on REGENXBIO’s gene therapy for Hurler syndrome after the discovery of a tumor in a patient, as well as to the action the FDA took against Sarepta following the Elevidys-related death. The agency is trying to balance safety and efficacy with “very robust pharmacovigilance,” Mohanty said. “FDA is a good partner generally,” Michael Meyers, head of M&A and Strategic Advisory Services at H.C. Wainwright, added. “I strongly believe that each trial, each application, is reviewed on the basis of that application without real political intervention, and it’s done at the reviewer level most of the time, without intervention at the director level or even above that,” Meyers continued. “I believe the regulators want to work with the cell and gene therapy companies.” Duncan agreed with Meyers’ assessment but said the FDA needs to be more accessible. “And that is where we are seeing some changes that are on the positive side. We are seeing them opening up communication pathways. We are seeing them open up pilot programs and allow for that.” But the panelists also agreed that improvements could be made, pointing especially to Japan as an example of effective regulatory communication. Nanoscope recently received Sakigake and Orphan Drug designations from Japan’s Ministry of Health, Labour and Welfare (MHLW) for MCO-010, its lead asset intended to treat patients with severe vision loss from inherited retinal diseases. “I never had such regulatory interaction, where almost every alternate day they were giving feedback through emails, not formal meetings and stuff,” Mohanty said. Duncan had a similar experience. In November 2023, Arcturus and partner CSL won approval in Japan for ARCT-154, the world’s first self-amplifying mRNA COVID-19 vaccine. Her experience working with the country’s Pharmaceuticals and Medical Devices Agency (PMDA) involved “continuous engagement, continuous feedback that allowed us to really progress approval there rather quickly,” she said. Historically, Duncan added, Japan has been particularly slow in terms of their regulatory process. “I don’t think they’re in that era any longer.” Editor’s note: BioSpace is a media partner of Phacilitate’s Advanced Therapies Week, held Feb. 9–12 at the San Diego Convention Center.

One day after bumping out CEO Paul McKenzie, Australia’s CSL revealed what he left behind: declining profit and a $1.1 billion hole due to lower US vaccine demand and increased generic competition. CSL announced McKenzie would retire immediately after the board “recognized he didn’t have the skills that we wanted for the future,” chairman Brian McNamee said Tuesday. The company has faced a tumultuous few years with failed spinoffs , headcount reductions and site closures . Net profit for the last six months of 2025 crashed by 81% and total revenue dropped 4% to $8.3 billion, CSL said Wednesday. The company will take $1.1 billion in impairment charges this year, most of it in the first half. Even with the headwinds, CSL maintained its guidance for fiscal 2026, projecting revenue growth of 2% to 3%. CSL was hit by declining vaccine demand in the US, particularly those for Covid-19, triggering a write-down of its 2022 licensing agreement with Arcturus Therapeutics, CFO Ken Lim said on Tuesday’s earnings call, according to an AlphaSense transcript. “We’ve seen a decline in Covid disease burden, as well as a significantly more onerous regulatory regime in the US, particularly regarding mRNA vaccines,” he said. Since Robert F. Kennedy Jr.’s appointment as HHS secretary last year, vaccine skepticism and pullback caused immunization rates to drop in the US, with vaccine makers struggling to generate profits. CSL’s vaccine business dropped 2% in the half-year that ended Dec. 31. The company is also facing impairment charges in its iron deficiency business called CSL Vifor. Its intravenous iron medication Venofer saw an uptick in generic competition last year, resulting in a reduced sales forecast. Even so, growth in nephrology assets offset iron demand decline, with CSL Vifor generating 12% more revenue for the last six months of 2025. Meanwhile, tighter plasma policy restrictions in China caused CSL’s plasma business to drop 7% in revenue, with a 27% hit for its blood plasma protein albumin for the six-month period. CSL is focused on growing its Chinese plasma footprint as a means to reduce imports into the country. “Our strategy for albumin is to continue to expand in China, and I think we’ll see a nice turnaround in the second half,” chief commercial officer Andy Schmeltz said Tuesday. The company also lost out on $170 million over its efforts to boost US manufacturing, leading other manufacturing assets to become “redundant,” the company said Wednesday. In November, CSL said it would invest $1.5 billion in the US.

Feb. 10, 2026 -- BaseLaunch, a platform partnering with scientists and entrepreneurs to launch, fund, and build early biotech ventures, announced today the pharmaceutical giant Pfizer has joined the Basel-based incubator, bringing the total number of leading pharma company partners to seven. Since its inception almost a decade ago, BaseLaunch has supported 27 biotech companies across various modalities and indications, with several of them having entered Phase II clinical trials. Adding Pfizer as a partner helps BaseLaunch further develop its portfolio companies, which have collectively raised over $1 billion (USD). The addition of Pfizer comes just two months after BaseLaunch announced its sixth global partner, proving the platform's value in fostering innovation. "BaseLaunch's roster of biotech heavy hitters continues to grow and adding new partnerships will significantly benefit our early-stage biotech ventures allowing for further growth and greater impact in the biotech industry," explained Stephan Emmerth, Director of BaseLaunch. Through its strategic investment in BaseLaunch, Pfizer gains access to proven quality life sciences deal flow in central Europe and BaseLaunch`s company-building capabilities, while also providing expertise in biotech innovation and development of medicines with promising drug development startups. Seven of the world's leading pharmaceutical companies are currently partners of BaseLaunch. In addition to Pfizer, these include AbbVie, Roche, Takeda, Novo Nordisk, Johnson & Johnson and CSL. The venture fund Pureos Bioventures is also a partner. The commitment of these prominent partners demonstrates the continued success of the BaseLaunch setup that also deeply embeds the portfolio companies in the Basel Area, a hotspot of European biopharma innovation. BaseLaunch is a Basel-based incubator that partners with scientists and entrepreneurs to help launch and grow exceptional biotech ventures developing cutting-edge therapeutics. The platform helps build companies from inception through to Series A funding, providing financing and supporting all aspects of company creation and growth. It is operationally run and financed by Basel Area Business & Innovation, the non-profit agency that focuses on promoting the Basel Area Life Sciences Supercluster, as Europe's most concentrated destination for biotech, pharma, healthtech, and medtech innovation. Domain and regional partners include KPMG, Vossius & Partner, Walder Wyss attorneys at law, WuXi AppTec, Lonza, Charles River Laboratories, Wuxi Biologics, Vischer, Hoffman&Partner, BioDuro, Catalent, as well as the Switzerland Innovation Park Basel Area. The content above comes from the network. if any infringement, please contact us to modify.