Pharmaceuticals

SHERIDAN, WYOMING – July 21, 2025 – Sarepta Therapeutics has entered a critical phase of uncertainty after reports surfaced that the U.S. Food and Drug Administration (FDA) is considering requesting a halt to all shipments of its Duchenne muscular dystrophy (DMD) gene therapy, Elevidys. The potential move follows the disclosure of a third patient death associated with the company's underlying gene therapy platform.

FDA Scrutiny Intensifies After Third Death Linked to Platform

SHERIDAN, WYOMING – July 21, 2025 – Bristol Myers Squibb (BMS) and Pfizer have announced a strategic move to offer their blockbuster blood thinner Eliquis directly to U.S. patients at a 40% discount off the current list price—marking a significant shift in their market approach ahead of Medicare’s negotiated pricing, which takes effect in 2026.

Starting September 8, eligible patients with a prescription will be able to purchase Eliquis through the expanded Eliquis 360 Support program. Originally designed for patient education and insurance assistance, the program now includes direct-to-consumer (DTC) sales aimed at reducing out-of-pocket expenses for uninsured, underinsured, and self-pay patients.

Strategic Expansion Amid Industry-Wide DTC Trends

The move reflects a growing trend among pharmaceutical companies to explore direct-to-consumer models. In recent years:

SHERIDAN, WYOMING – July 21, 2025 – Bristol Myers Squibb’s (BMS) leading anemia therapy, Reblozyl, has failed to meet its primary endpoint in a pivotal Phase III trial for myelofibrosis-associated anemia, highlighting the ongoing challenges in addressing this difficult-to-treat condition.

The INDEPENDENCE study, designed to evaluate Reblozyl's ability to reduce patients’ dependence on red blood cell transfusions, did not achieve statistical significance on its primary objective—independence from transfusions for 12 consecutive weeks within the first 24 weeks of treatment. The company disclosed the outcome in a news release last Friday but withheld detailed data on transfusion independence rates.

SHERIDAN, WYOMING – July 21, 2025 – GlaxoSmithKline’s ambitions to relaunch its antibody-drug conjugate Blenrep for multiple myeloma treatment faced a critical blow last Thursday, as the U.S. Food and Drug Administration’s Oncologic Drugs Advisory Committee (ODAC) rejected two proposed combination therapies over persistent safety and dosing concerns.

Safety Risks and Dosing Shortfalls Undermine Confidence

The advisory panel found that the benefits of Blenrep do not outweigh its risks for patients with relapsed or refractory multiple myeloma. In two separate votes, the committee expressed strong reservations about the drug’s risk-benefit profile:

SHERIDAN, WYOMING – July 21, 2025 – Sarepta Therapeutics has taken bold steps to reshape its business, unveiling a sweeping organizational overhaul that includes significant workforce reductions, a strategic pipeline pivot, and a new safety warning on its flagship gene therapy product. The move, announced late Wednesday, is positioning the company for long-term resilience — a shift that has already caught the attention of analysts and investors alike.

Strategic Restructuring Targets Operational Focus

Sarepta confirmed it has laid off over one-third of its workforce, parting ways with approximately 500 employees. This downsizing accompanies a pivot toward its siRNA platform assets, a strategic move designed to streamline operations and sharpen the company’s research focus. The restructuring coincided with second-quarter financial disclosures, revealing $513 million in sales from Sarepta’s Duchenne muscular dystrophy (DMD) portfolio.

SHERIDAN, WYOMING – July 21, 2025 – Novartis is intensifying its strategic push to relocate key drug manufacturing operations to the United States, aiming to safeguard its U.S. market position against potential tariff impacts. In its second-quarter earnings call last Thursday, CEO Vas Narasimhan emphasized the urgency of reshoring production, while acknowledging the complexity of fully transitioning manufacturing operations.

Novartis Targets Full U.S. Production for Key Medicines

Novartis confirmed its commitment to moving the production of its major medicines for the American market entirely to the U.S. “We’re moving as fast as possible to ensure that within the next few years we’re able to produce our key medicines [in the U.S.] and therefore fully mitigate the tariff impact,” stated Narasimhan.

SHERIDAN, WYOMING – July 12, 2025 – Moderna has received full approval from the U.S. Food and Drug Administration for its COVID-19 vaccine, Spikevax, for children aged 6 months through 11 years who are at higher risk of contracting the disease, reinforcing the company’s position as a key innovator in the mRNA vaccine space.

Spikevax, which had previously been available to children only under an emergency use authorization, is now fully approved for:

• Children 6 months through 11 years at increased risk
• Individuals aged 6 months through 64 years at increased risk
• All adults 65 years and older

Moderna expects to make the updated Spikevax shot available ahead of the 2025-2026 respiratory virus season.

Momentum Across Moderna’s mRNA Vaccine Pipeline

SHERIDAN, WYOMING – July 12, 2025 – A landmark release of 200 complete response letters (CRLs) by the U.S. Food and Drug Administration (FDA) has unveiled a decade of regulatory decisions behind once-rejected therapies, now approved and on the market. Among the most revealing cases are Eli Lilly’s Alzheimer’s treatment Kisunla, Sarepta’s Duchenne muscular dystrophy drug Vyondys 53, and Gilead’s long-acting HIV therapy Sunlenca.

This unprecedented act of “radical transparency” by the FDA marks a rare window into the regulatory reasoning typically hidden from public view. Each letter provides detailed insight into the challenges biopharmaceutical companies face—from inadequate safety data to packaging compatibility issues—offering strategic guidance for future drug development and market approval.

Kisunla CRL Highlights FDA’s Long-Term Safety Expectations

SHERIDAN, WYOMING – July 8, 2025 – A coalition of major U.S. medical societies has filed a federal lawsuit against Health and Human Services Secretary Robert F. Kennedy Jr., challenging his directive to remove COVID-19 vaccines from routine immunization schedules for healthy children and pregnant women.

The lawsuit, filed Monday in the U.S. District Court for Massachusetts, responds to Kennedy’s May announcement eliminating these groups from federal COVID-19 vaccine guidelines. The plaintiffs—among them the American Academy of Pediatrics, the American College of Physicians, and the Infectious Diseases Society of America—are seeking immediate legal intervention to reverse the decision.

Medical organizations cite danger to vulnerable populations

SHERIDAN, WYOMING – July 8, 2025 – The newly signed One Big Beautiful Bill from President Donald Trump is poised to reshape the U.S. pharmaceutical policy landscape, restoring expanded orphan drug exemptions from the Inflation Reduction Act (IRA) while introducing significant Medicaid cuts and dropping regulatory reforms on pharmacy benefit managers (PBMs). The bill signals a strategic shift that may benefit the biopharma sector, while raising concerns about access and affordability in public healthcare.

Expanded orphan drug exemptions bolster rare disease R&D

Among the most consequential elements for the pharmaceutical industry is the reinstatement of broader protections for orphan drugs from Medicare price negotiations. The IRA had previously limited exemptions to drugs with only a single rare disease indication, but under the new law, those protections will now extend across multiple indications.