Novo Nordisk Submits CagriSema for FDA Review as a Once-weekly GLP-1–amylin Combination Therapy for Weight Management
Novo Nordisk has officially filed a New Drug Application (NDA) with the U.S. FDA for CagriSema, a once-weekly subcutaneous injection intended for chronic weight management. CagriSema represents a significant technological leap in the incretin space, combining the established GLP-1 receptor agonist semaglutide (the active ingredient in Wegovy) with cagrilintide, a long-acting amylin analog. While semaglutide primarily targets appetite suppression and gastric emptying through the GLP-1 pathway, cagrilintide acts on the amylin receptors to enhance satiety and delay stomach emptying via a distinct mechanism. This dual-action approach aims to achieve weight-loss outcomes that surpass the current “gold standard” set by monotherapies.
The findings from the REDEFINE clinical trial program primarily support the submission. In the REDEFINE 1 Phase 3 trial, participants treated with CagriSema achieved a mean weight loss of approximately 25% over 68 weeks, a result that notably exceeds the roughly 15-17% typically observed with semaglutide alone. Crucially, the trial also demonstrated that CagriSema was superior to cagrilintide alone in reducing body weight, suggesting a synergistic effect between the two molecules. The safety profile remained consistent with the incretin class, with gastrointestinal events (nausea, diarrhea, and vomiting) being the most frequently reported adverse effects, generally occurring during the dose-escalation phase.
If approved, CagriSema will be Novo Nordisk’s primary weapon against Eli Lilly’s Zepbound (tirzepatide), which utilizes a GLP-1/GIP dual-agonist approach. By targeting the amylin pathway, Novo Nordisk is betting that a multi-hormonal strategy can push medical weight loss toward the 25-30% range, previously achievable only through bariatric surgery. This filing marks a pivotal moment in the “obesity wars,” as manufacturers race to provide more potent, efficacious, and tolerable options for a global population increasingly burdened by metabolic disease.
SAPHNELO Gains EU Approval for at-home Subcutaneous Dosing with a Pre-filled Pen in SLE
The European Commission has expanded the marketing authorization for AstraZeneca’s SAPHNELO (anifrolumab) to include a new subcutaneous (SC) administration route via a pre-filled pen. SAPHNELO is a first-in-class type I interferon (IFN) receptor antagonist indicated as an add-on therapy for adult patients with moderate to severe, autoantibody-positive systemic lupus erythematosus (SLE). Previously, the drug was only available as an intravenous (IV) infusion, requiring patients to visit a clinic or infusion center every four weeks. The approval of the subcutaneous pen transforms the treatment landscape for SLE by allowing for self-administration at home, significantly reducing the logistical burden on patients and the healthcare system.
The approval is supported by data from the Phase 3 Orchid and Tulip programs, as well as the PALOMA study, which specifically evaluated the subcutaneous formulation. These trials demonstrated that subcutaneous delivery of anifrolumab achieved pharmacokinetic and pharmacodynamic results comparable to those of the IV infusion. Importantly, the efficacy in reducing lupus disease activity, measured by the British Isles Lupus Assessment Group-based Composite Lupus Assessment (BICLA) and the SLE Responder Index (SRI-4), was maintained across the different delivery methods. The safety profile for the subcutaneous pen was also favorable, with the most common adverse reactions being upper respiratory tract infections and bronchitis, alongside expected mild injection-site reactions.
This regulatory milestone is a significant win for patient autonomy in the lupus community. SLE is a chronic, often unpredictable disease that predominantly affects women of childbearing age, many of whom manage professional and family responsibilities alongside their condition. Transitioning from a 30-minute clinic-based infusion to a once-weekly or once-monthly (depending on the specific regimen) at-home injection provides greater flexibility and can improve long-term adherence. For AstraZeneca, this delivery expansion helps solidify SAPHENEO’s competitive position against other biologics, such as GSK’s Benlysta, which has long offered both IV and SC options.
Cytokinetics Receives FDA Approval for MYQORZO in Adults with Symptomatic Obstructive Hypertrophic Cardiomyopathy
The U.S. FDA has granted approval to Cytokinetics’ MYQORZO (aficamten) for the treatment of adults with symptomatic obstructive hypertrophic cardiomyopathy (oHCM). MYQORZO is a next-generation cardiac myosin inhibitor designed to reduce the hypercontractility that characterizes HCM. In patients with the obstructive form of the disease, the heart muscle becomes excessively thick, physically blocking blood flow out of the left ventricle. By selectively inhibiting the cardiac myosin ATPase, MYQORZO reduces the number of active actin-myosin cross-bridges, thereby relaxing the heart muscle, relieving the obstruction, and improving the heart’s overall filling pressure.
The approval is centered on the results of the pivotal Phase 3 SEQUOIA-HCM trial. The study met its primary endpoint, showing a statistically significant and clinically meaningful increase in peak oxygen consumption (pVO2) compared to placebo. Patients treated with aficamten also showed rapid and sustained improvements in New York Heart Association (NYHA) functional class and Kansas City Cardiomyopathy Questionnaire (KCCQ) scores, reflecting better physical capacity and quality of life. A key differentiator for MYQORZO is its relatively short half-life, which enables faster dose titration and potentially a more manageable safety profile regarding the risk of reducing the left ventricular ejection fraction (LVEF) too severely.
MYQORZO enters the hypertrophic cardiomyopathy market as a direct competitor to Bristol Myers Squibb’s CAMZYOS (mavacamten). While Camzyos was a breakthrough as the first-in-class myosin inhibitor, it carries a heavy Risk Evaluation and Mitigation Strategy (REMS) program due to the risk of heart failure. Cytokinetics has emphasized that aficamten’s wider therapeutic window and predictable pharmacokinetics might offer a more streamlined experience for both clinicians and patients. This approval is a transformative event for Cytokinetics, transitioning the company into a commercial-stage entity and providing a new, high-value treatment option for the thousands of patients suffering from the debilitating breathlessness and fatigue associated with oHCM.
Boehringer’s JASCAYD Receives U.S. FDA Approval for Adult Progressive Pulmonary Fibrosis
Boehringer Ingelheim has received FDA approval for JASCAYD (neridamstat), a novel oral therapy for the treatment of adults with progressive pulmonary fibrosis (PPF). Pulmonary fibrosis involves the irreversible scarring of lung tissue, making breathing increasingly difficult as the lungs lose elasticity. While Boehringer already markets Ofev (nintedanib) for progressive pulmonary fibrosis treatment, JASCAYD represents a new therapeutic class targeting the specific cellular signaling pathways that drive the “progressive” nature of the scarring, regardless of the underlying clinical diagnosis (such as autoimmune-related ILD or idiopathic disease).
The FDA’s decision was informed by data from the Phase 3 FIBRONEER-ILD trial. The study demonstrated that JASCAYD significantly slowed the decline in Forced Vital Capacity (FVC), the standard measure of lung function, over 52 weeks compared with placebo. Notably, JASCAYD demonstrated efficacy as both a monotherapy and in combination with existing background antifibrotic treatments. The safety data indicated that the most common adverse events were gastrointestinal in nature. However, the incidence of severe liver enzyme elevations was lower than that observed with some earlier-generation antifibrotics. This favorable safety profile is critical, as patients with PPF often require lifelong medication and may already be on multiple other therapies for comorbid conditions.
With JASCAYD’s approval, Boehringer Ingelheim strengthens its dominant position in the interstitial lung disease (ILD) market. The “progressive” phenotype of pulmonary fibrosis is particularly challenging because it implies that, despite standard treatment, the patient’s lung function continues to deteriorate. JASCAYD provides a new mechanism of action to intervene in this destructive cycle. For the medical community, this approval offers a much-needed second-line or alternative option for patients who do not tolerate current treatments or who require a more aggressive multi-pathway approach to stabilize their respiratory health.
BioMarin to Acquire Amicus Therapeutics for $4.8B, Enhancing Rare Disease Leadership
In a major move to consolidate leadership in the rare disease sector, BioMarin Pharmaceutical has entered into a definitive agreement to acquire Amicus Therapeutics for approximately $4.8 billion in an all-cash transaction. This strategic acquisition unites two of the most prominent players in the lysosomal storage disorder (LSD) space. BioMarin is best known for its portfolio of enzyme replacement therapies and its recently approved gene therapy for hemophilia A, while Amicus has built a successful franchise around Galafold (migalastat) for Fabry disease and the newly launched Pombiliti plus Opfolda for Pompe disease.
The deal is driven by several key synergies. First, the acquisition immediately bolsters BioMarin’s revenue stream with Galafold, which is a market-leading oral chaperone therapy with a strong global footprint. Second, the addition of the Pombiliti/Opfolda combination gives BioMarin a high-growth asset in the Pompe disease market, a segment where they previously lacked a dominant presence. Beyond the immediate commercial gains, BioMarin is interested in Amicus’s specialized protein engineering capabilities and its pipeline of next-generation gene therapies. By folding Amicus’s R&D engine into its own, BioMarin aims to accelerate the development of curative treatments for ultra-rare metabolic and genetic conditions.
The $4.8 billion price tag reflects a significant premium over Amicus’s recent trading price, signaling BioMarin’s confidence in the long-term value of the Amicus portfolio. Analysts view this as a defensive yet aggressive move in a landscape where large pharmaceutical companies (such as Sanofi and Takeda) are also expanding their rare-disease footprints. For BioMarin, the acquisition helps diversify its risk away from its high-stakes gene therapy launches. It creates a more robust, diversified rare-disease powerhouse with multiple “blockbuster”- potential products across different stages of the lifecycle. The transaction is expected to close in the first half of 2026, pending customary regulatory approvals and shareholder consent.
