Drug pipeline for August 2026

At Prime Therapeutics (Prime), we have positioned ourselves to best prepare our clients to manage new drugs. Our clinical and trade relations teams keep a keen eye on drugs awaiting approval by the Food and Drug Administration (FDA).

 

August 2026: garetosmab

Regeneron Pharmaceutical’s garetosmab is under Priority Review by the FDA for the treatment of fibrodysplasia ossificans progressiva (FOP), an ultra-rare progressive genetic disorder of the connective tissues. The FDA also granted Fast Track and Orphan Drug designations. Garetosmab is a fully human, monoclonal antibody that binds and neutralizes Activin A, a protein involved in the development of heterotopic ossification (HO) associated with FOP. Topline results from the Phase 3 OPTIMA trial demonstrated that intravenous (IV) garetosmab administered every four weeks significantly reduced the formation of new HO lesions (primary endpoint) compared with placebo in adults with FOP and confirmed type I Activin A receptor (ACVR1) mutation.¹‧² At 56 weeks, the garetosmab 3 mg/kg dose reduced new HO lesions by 94% compared to placebo (one versus 19 lesions; P=0.0274), while the 10 mg/kg dose resulted in a 90% reduction (two versus 19 lesions; P=0.026). If approved, garetosmab may compete with the retinoid palovarotene (Sohonos) for treatment of adults with FOP, but Sohonos may have advantages over garetosmab due to Sohonos’ oral administration and established use in the pediatric population.

 

Aug. 2, 2026: vusolimogene oderparepvec (RP1)

Replimune Group has resubmitted RP1, an engineered herpes simplex virus type 1 (HSV-1) oncolytic therapy, for use in combination with nivolumab (Opdivo) for the treatment of advanced melanoma that has progressed on anti-PD-1 treatment. RP1 is administered by intratumoral injection every two weeks for eight cycles, with Opdivo administered intravenously for 30 cycles. This is the third review for RP1 after the FDA issued Complete Response Letters (CRLs) in July 2025 and April 2026, concluding that the clinical evidence supporting the application was insufficient. The recent resubmission is seeking Accelerated Approval for RP1. Data from the Phase 2 IGNYTE study was presented at the American Society of Clinical Oncology (ASCO) Annual Meeting in May 2026.³ It revealed that, at a median follow-up of three years, RP1 resulted in a median overall survival (OS) of 32.2 months. OS rates at one, two and three years of follow-up were 75.3%, 61.6% and 45.5%, respectively. In addition, the three-year OS rate was 81.8% among responders compared with 22.5% for nonresponders. The FDA has granted RP1 Breakthrough Therapy designation and the FDA’s Cellular, Tissue, and Gene Therapies Advisory Committee will review data on its safety and efficacy on July 30, 2026. If approved, RP1 plus Opdivo will offer a new, second-line option for advanced melanoma, an area with limited available treatments.

For more information, see the vusolimogene oderparepvec (RP1) Deep dive in the April 2025 edition of Prime’s Quarterly Drug Pipeline.

 

Aug. 5, 2026: trivalent influenza mRNA vaccine (mRNA-1010; mFlusiva)

Moderna is seeking full approval for their seasonal mRNA vaccine mRNA-1010 for adults 50–64 years of age and Accelerated Approval for adults 65 years and older. A two-season, Phase 3 study (NCT06602024) randomized 40,703 participants 50 years and older to receive a single, intramuscular injection of mRNA-1010 (37.5 μg) or a licensed standard-dose comparator.⁴ The primary endpoint was a reverse-transcriptase-polymerase-chain-reaction (RT-PCR)-confirmed, protocol-defined, influenza-like illness from at least 14 days after vaccination through the end of the influenza season. mRNA-1010 was found to be noninferior to comparator vaccines ― the primary endpoint was observed in 2% of mRNA-1010 recipients compared with 2.8% of comparator recipients, corresponding to a relative vaccine efficacy (rVE) against influenza illness of 26.6% for mRNA-1010 in the overall study population. Strong rVE was reported for individual influenza strains contained in the vaccine, including A/H1N1 (rVE=29.6%), A/H3N2 (rVE=22.2%) and the B/Victoria lineages (rVE=29.1%). Among participants 65 years of age or older, the rVE was 27.4%. In June 2026, The FDA’s Vaccines and Related Biological Products Advisory Committee voted unanimously (9–0) that the benefits of mRNA-1010 outweigh the risk in adults 50–64 years of age and adults 65 years of age and older. If approved, mRNA-1010 will be the first mRNA-based seasonal influenza vaccine in the United States.

 

Aug. 17, 2026: iberdomide

Bristol Myers Squibb’s first-in-class cereblon E3 ligase modulator, iberdomide, is under FDA Priority Review for use in combination with daratumumab plus dexamethasone for the treatment of relapsed or refractory multiple myeloma (RRMM). The FDA granted iberdomide Breakthrough Therapy designation for the proposed indication. The open-label, Phase 3 EXCALIBER-RRMM study is evaluating iberdomide, in combination with daratumumab and dexamethasone, compared with a combination of daratumumab, bortezomib and dexamethasone for RRMM.⁵ Stage 1 of the study identified the optimal dose of iberdomide as 1 mg. Topline results from the randomized, Stage 2 portion of the study have not been publicly announced. The FDA accepted the application based on results from a planned analysis of minimal residual disease negativity rates. If approved, iberdomide will provide a new mechanism of action to combat RRMM.

 

Aug. 22, 2026: deramiocel

Nippon Shinyaku and Capricor Therapeutics are seeking full FDA approval for deramiocel, an off-the-shelf cellular therapy consisting of allogeneic cardiosphere-derived cells, for the treatment of Duchenne muscular dystrophy (DMD) cardiomyopathy. DMD is a rare, X-linked neuromuscular disorder associated with progressive muscle degeneration, pulmonary dysfunction and cardiomyopathy. Deramiocel was granted Orphan Drug, Rare Pediatric Disease (RPD) and Regenerative Medicine Advanced Therapy (RMAT) designations. In July 2025, the FDA issued a CRL for deramiocel, requiring additional clinical data; however, review of the Biologic License Application was resumed after additional data from the Phase 3 HOPE-3 trial was submitted. Topline results from the study revealed that deramiocel 1.5×10⁸ cells administered by IV infusion every 3 months reduced the decline in upper-limb function by 54% (P=0.029) at 12 months compared with placebo, as based on the Performance of Upper Limb (PUL v2.0)⁶ primary endpoint measure. Deramiocel also slowed cardiomyopathy progression by 91% (P=0.04), as measured by left ventricular ejection fraction (LVEF). If approved, deramiocel will be the first cellular therapy to treat DMD cardiomyopathy, a leading cause of death among patients with DMD.

For more information, see the deramiocel Deep dive in the April 2025 edition of Prime’s Quarterly Drug Pipeline.

 

Aug. 23, 2026: pariglasgene brecaparvovec (DTX401)

The FDA granted a Priority Review for DTX401 for the treatment of glycogen storage disease type 1a (GSDIa), a rare, life-threatening genetic disorder caused by an inborn error of carbohydrate metabolism, with characteristic features of severe hypoglycemia and liver and kidney disease. Patients with GSDIa must avoid fasting and consume prescribed amounts of cornstarch at frequent intervals throughout the day and night to maintain adequate glucose levels. DTX401 is an adeno-associated virus vector type 8 gene therapy by Ultragenyx designed to provide stable expression and activity of glucose-6-phosphatase (G6Pase) and address the underlying cause of the disease. The therapy has received Fast Track, Orphan Drug, RPD and RMAT designations from the FDA. In the Phase 3 GlucoGene crossover trial, a single IV infusion of DTX401 (1×10¹³ genome copies/kg) significantly reduced daily cornstarch requirements at Week 48 compared with placebo (mean reduction, 41% versus 10%, respectively; P<0.0001).⁷ At Week 96, patients treated with DTX401 achieved a 61% reduction in cornstarch consumption from baseline, with a low incidence of hypoglycemia. If approved, DTX401 would be the first pharmacologic therapy to target the underlying genetic cause of GSD1a.

For more information, see the pariglasgene brecaparvovec (DTX401) Deep dive in the April 2026 edition of Prime’s Quarterly Drug Pipeline.

 

All brand names are property of their respective owners.

References