FDA Shake Up, Sarepta Rebounds, and Trump Targets Drug Prices — This Week in Biotech #63
Prasad’s resignation boosts biotech stocks, Sarepta regains ground, and Trump demands global price matching from pharma CEOs (July 25–31, 2025).
Hi and welcome to This Week in Biotech by Biotech Blueprint, edition 63, covering biotech & pharma news from July 25th to July 31st, 2025.
🎙️ Biotech Blueprint brings you weekly video updates on the latest biotech and pharma news, plus in-depth podcast interviews with industry leaders. You can find us on YouTube, Spotify, and Apple Podcasts.
In the latest episode, I teamed up with the Biotech Capital Compass to break down Sarepta Therapeutics’s recent safety crisis, and what the FDA’s partial green light means for Elevidys, investor confidence, and the broader AAV field. We dig into the science, regulatory dynamics, and what might come next.
And the accompanying article published on Subsatck:
THIS WEEK IN BIOTECH VIDEO SUMMARY
THIS WEEK’S KEY TAKEAWAYS 🔑
FDA leadership change signals softer stance? The sudden resignation of FDA biologics chief Vinay Prasad, known for his tough stance on accelerated approvals, triggered a sharp rebound in biotech stocks like Replimune (+100%), Capricor, and Sarepta. His departure, following controversy over Sarepta’s Elevidys gene therapy, may mark a regulatory pivot toward greater flexibility for gene therapies and rare disease drugs.
Sarepta’s wild week: The FDA allowed ambulatory use of Elevidys to resume. Sarepta surged 38% on the news. Analysts are split. Some see renewed momentum, while others warn of liquidity risk and international headwinds.
Trump cracks down on drug prices: President Trump issued demands to 17 pharma giants to implement “most favored nation” pricing within 60 days. The policy ties U.S. Medicaid pricing to the lowest international prices and threatens aggressive enforcement. The move rattled markets and sparked backlash.
AbbVie eyes psychedelics: AbbVie is reportedly in talks to acquire Gilgamesh Pharmaceuticals for $1B. The news boosted psychedelic stocks.
Two notable FDA approvals:
Apellis’ Empaveli became the first FDA-approved treatment for C3 glomerulopathy and IC-MPGN, two rare and progressive kidney diseases. Backed by strong data, this approval could be a commercial and clinical inflection point in nephrology.
PTC’s Sephience was approved for phenylketonuria (PKU), including use in infants as young as one month. It restores BH4 to lower phenylalanine levels and marks a major step forward for this rare disease community.
Moderna and Pfizer prep for COVID season: Both received positive EU recommendations for updated COVID-19 vaccines targeting LP.8.1. Moderna secured full EU approval, even as it announced a 10% workforce cut due to soft vaccine demand. The company is now pivoting toward oncology and rare diseases.
Clinical Standouts:
Eli Lilly’s Jaypirca beat Imbruvica in a head-to-head CLL trial, showing a clear edge in response rate and tolerability, especially in newly diagnosed patients.
Cardiff Oncology’s onvansertib showed promise in a phase 2 colorectal cancer trial, but shares dropped 26%.
MARKET UPDATES
🔹 Vinay Prasad’s sudden resignation as head of the FDA’s biologics division has triggered a wave of investor optimism, particularly for companies recently hit by regulatory pushback. Known for his skepticism of accelerated approvals and reliance on surrogate endpoints, Prasad had only been in the role for a few months. His departure comes on the heels of controversy around Sarepta’s Elevidys gene therapy and may signal a shift in tone at the agency. Stocks like Replimune, Capricor, and Sarepta surged following the news, with Replimune gaining over 100% after analysts suggested Prasad may have played a key role in its recent FDA rejection despite prior trial alignment and a Breakthrough Therapy designation. Some now see a path for regulatory reconsideration, especially if new leadership proves more open to innovation.
BIOTECH/PHARMA NEWS 🧬
🔹 Another tumultuous week for Sarepta
Last week’s recap: Sarepta reported a third patient death tied to its AAV platform, this time in a limb-girdle muscular dystrophy trial, prompting the FDA to request a halt to Elevidys shipments. Sarepta complied, paused U.S. distribution, and agreed to add a black box warning. The FDA also placed a clinical hold on all LGMD trials and revoked Sarepta’s AAVrh74 platform designation. Roche paused international shipments in FDA-dependent markets.
This week, Sarepta received its first piece of good news. On July 29, the FDA concluded that the death of an 8 year old Brazilian boy was unrelated to Elevidys, and recommended lifting the voluntary hold, but only for ambulatory patients. Non-ambulatory patients remain on pause, and new studies will likely be required to support broader access.
Markets reacted quickly. Sarepta’s stock jumped 38% premarket, and analysts at Oppenheimer upgraded the stock to Outperform, citing renewed confidence in the Elevidys risk-benefit profile. J.P. Morgan also moved its rating from Underweight to Neutral but warned that regaining physician, patient, and investor trust may take time, and that headline risk still looms.
That optimism wasn’t universal. Barclays downgraded Sarepta to Underweight, raising liquidity concerns tied to debt, milestone obligations, and access to its $600M credit revolver. Meanwhile, the European Medicines Agency issued a negative opinion on Elevidys, further clouding its international prospects.
On a brighter note, Sarepta’s RNAi partner Arrowhead earned a $100M milestone after hitting dosing targets in a myotonic dystrophy trial, a reminder that Sarepta’s pipeline goes beyond AAV. But the core challenge remains: demonstrating that its AAV platform can be used safely and consistently, particularly in fragile patients.
Then on July 30, the story took a sharp political turn. Dr. Vinay Prasad, head of the FDA’s biologics division and the person behind the agency’s recent Elevidys actions, abruptly resigned. The FDA has named George Tidmarsh, a recent appointee and former biotech executive, as acting head of CBER.
Prasad’s departure followed mounting criticism from conservative commentators and patient advocacy groups. According to STAT and the AP, his position became untenable after pausing Elevidys trials and shipments, a move he defended based on safety concerns, but which triggered fierce backlash from parents, biotech leaders, and media outlets. In response, the FDA partially relented, allowing ambulatory use to resume.
Prasad had long expressed skepticism toward Sarepta’s AAVrh74 platform and the FDA’s past flexibility with gene therapy approvals. But his caution clashed with growing political and patient pressure for broader access, especially under an administration prioritizing speed and autonomy in medical decisions. Analysts now say Prasad’s exit could represent a shift toward a more permissive tone for gene therapy and rare disease reviews, potentially benefiting Sarepta and other developers navigating an uncertain regulatory landscape.
🔹 President Donald Trump has sent strongly worded letters to 17 pharmaceutical companies, including Pfizer, Merck, Eli Lilly, Johnson & Johnson, Gilead, Novartis, and AbbVie, demanding that they implement most favored nation (MFN) pricing within 60 days. Under his May 2025 executive order, U.S. drug prices for Medicaid must match the lowest prices charged in other developed countries, and newly launched drugs must also be offered at those levels. Firms are directed to offer direct to consumer pricing for high volume drugs, repatriate excess overseas drug revenues back to U.S. patients and taxpayers, and make binding commitments by Sept. 29, 2025. Trump warned that failing to comply would trigger the administration’s use of “every tool in our arsenal.” The announcement caused stock declines of around 2 to 4%, and triggered pushback from industry groups like PhRMA and BIO, which argued the policy could undermine U.S. pharmaceutical innovation.
🔹 AbbVie is reportedly in talks to acquire Gilgamesh Pharmaceuticals, a privately held biotech focused on next-gen psychedelic therapies for psychiatric conditions. The potential $1B deal would build on a partnership announced in May and comes amid AbbVie’s broader M&A spree following Humira’s patent loss. Gilgamesh recently reported promising mid-stage results for its lead candidate in depression, and the news sparked a rally in psychedelic stocks. Analysts say the move could further validate investor interest in the space, especially under a more supportive U.S. regulatory climate.
🔹 The FDA has approved Apellis’ Empaveli (pegcetacoplan) as the first treatment for C3 glomerulopathy (C3G) and primary IC-MPGN in patients aged 12+. The approval is based on phase 3 data showing a 68% reduction in proteinuria, stable kidney function, and significant clearance of C3 deposits. Empaveli’s broad label includes both native and post-transplant disease and reflects its potential to prevent kidney failure in these rare and severe conditions. This marks Apellis’ third FDA approval in four years and expands the role of C3-targeted therapies in nephrology.
🔹 PTC Therapeutics has secured FDA approval for Sephience (sepiapterin), a new treatment for phenylketonuria (PKU) that’s now cleared for use in both adults and children as young as one month. The approval includes broad labeling across PKU subtypes responsive to sepiapterin and follows strong phase 3 data showing durable reductions in blood phenylalanine levels. Sephience works by restoring BH4, a key cofactor needed to break down phenylalanine. Already authorized in Europe, the drug marks a significant milestone for the PKU community, where treatment options remain limited. PTC plans an immediate U.S. launch and is pursuing approvals in additional countries.
🔹 The FDA has approved Ascendis Pharma’s once-weekly growth hormone therapy Skytrofa for adults with growth hormone deficiency, marking the product’s second major indication after its 2021 pediatric approval. The decision was supported by positive phase 3 data showing strong efficacy and safety compared to both placebo and daily somatropin. Skytrofa’s weekly dosing schedule is designed to ease treatment burden and improve adherence, addressing a challenge with daily injections. Trials targeting conditions such as Turner syndrome, SHOX deficiency, and achondroplasia are expected to begin later this year.
🔹 On July 25, Gilead’s lenacapavir received a positive CHMP opinion as the first-ever twice-yearly injectable HIV PrEP option, setting it up for potential approval across the EU. If greenlit, it could reshape HIV prevention access in both Europe and low- and middle-income countries through WHO-linked pathways. Meanwhile, KalVista secured its own CHMP nod for sebetralstat, which could become the first oral on-demand treatment for hereditary angioedema (HAE) attacks in Europe. Backed by the largest HAE trial to date, the drug promises faster relief without the need for injections. Both decisions now head to the European Commission, with approvals expected by year-end for lenacapavir and by early October for sebetralstat.
🔹 Both Moderna and Pfizer/BioNTech both received positive CHMP recommendations for their LP.8.1-adapted COVID-19 vaccines, now authorized for individuals as young as six months across the EU ahead of the 2025/2026 respiratory season. The European Commission has since granted Moderna full marketing authorization, paving the way for rollout of its updated Spikevax formulation across all EU member states and select EEA countries. However, Moderna’s momentum is tempered by internal restructuring as the company announced a 10% workforce reduction as part of a broader cost-cutting plan, citing weaker than expected vaccine demand and delays in its combination COVID-flu program. CEO Stéphane Bancel reaffirmed plans for up to eight new approvals in the next three years as the company shifts focus to oncology and rare diseases. Meanwhile, Pfizer/BioNTech’s updated Comirnaty vaccine remains under final EC review, but manufacturing is already underway to ensure supply readiness.
CLINICAL TRIAL UPDATES 📊
🔹 Cardiff Oncology reported encouraging interim results from its phase 2 trial of onvansertib combined with standard chemotherapy in first-line RAS-mutated metastatic colorectal cancer. The 30mg dose arm achieved a 49% confirmed objective response rate versus 30% in the control group, along with signs of improved progression-free survival and deeper tumor responses. The drug was well-tolerated, with no unexpected safety signals. Despite the positive data, shares of Cardiff fell 26%. The company expects to share a program update in Q1 2026 as it advances toward a registrational trial.
🔹 Eli Lilly’s Jaypirca (pirtobrutinib), the first approved non-covalent BTK inhibitor, outperformed J&J’s and AbbVie’s Imbruvica (ibrutinib) on response rate in the phase 3 BRUIN CLL-314 trial for chronic lymphocytic leukemia (CLL) and small lymphocytic lymphoma (SLL). The study met its primary endpoint, showing not just non-inferiority but a statistically significant advantage for Jaypirca. Progression-free survival data were still maturing but showed a favorable trend. Notably, this is the first head-to-head BTK inhibitor study in CLL to include treatment-naïve patients, where pirtobrutinib showed the most pronounced benefit. With a tolerability profile consistent with earlier trials, Jaypirca continues to strengthen its case for broader use across the CLL/SLL treatment landscape.
AI SUMMARY 🎙️
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DISCLOSURE: I have no business relationships with any company mentioned in this article.