^{Maryam Tabatabai, PharmD, is the associate vice president of clinical information at Prime Therapeutics.}  

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Following a year of notable progress in the drug pipeline, 2026 is poised to accelerate the arrival of new therapies across a range of chronic, rare and complex diseases. 

U.S. Food and Drug Administration (FDA) Commissioner Marty Makary, M.D., MPH, has emphasized the agency’s focus on approving faster cures and meaningful treatments backed by strong data. Makary has signaled a desire in the next one to two years to see breakthroughs in several critical areas, including type 1 diabetes, neurodegenerative disorders and early-stage cancers that could be treated without surgery, chemotherapy or radiation. He has also highlighted the need for a universal flu vaccine that eliminates yearly shots as well as faster pathways for potentially promising post-traumatic stress disorder (PTSD) treatments. 

Against this backdrop, the FDA — which regulates products for 20% of the U.S. economy and this year marks its 120th anniversary — is preparing for a wave of decisions in the beginning to middle of the year that could introduce new treatment options for dry eye disease, rare disease and type 2 diabetes — particularly once-weekly basal insulin therapies. Building on the foundation laid in 2025, the FDA has issued a new position on a one-trial requirement for approval of novel products, instead of two trials, and launched a framework for approval of targeted bespoke individualized therapies especially for ultra-rare diseases. Over time, the agency is also expected to further eliminate or reduce animal testing using New Approach Methodologies or NAMs data (such as artificial intelligence-based computational modeling and organ-on-a-chip technology) as it implements its roadmap in preclinical safety studies. Safety and efficacy still continue to be important factors for drug approval.  

Here are 11 of the major decisions Prime Therapeutics (Prime) expects from the FDA in early 2026 .  

1. Novo Nordisk’s high-dose subcutaneous glucagon-like peptide-1 (GLP-1) receptor agonist medication semaglutide (Wegovy) for obesity treatment. With an anticipated decision in Q1 2026, this 7.2 mg strength showed 18.7% weight loss versus 2.4 mg injectable Wegovy, offering another once-weekly option for adults with obesity. 

2. Janssen’s subcutaneous teclistamab-cqyv (Tecvayli) for relapsed/refractory multiple myeloma in combination with daratumumab/hyaluronidase-fihj (Darzalex). With an anticipated decision in Q1 2026, this drug could establish an off‑the‑shelf bispecific antibody–based regimen for relapsed or refractory multiple myeloma, which has shown significant improvement in progression-free survival and overall survival compared with standard therapies, expanding access to deep, durable responses without the need for individualized cell therapy.  

3. Eli Lilly’s oral GLP-1 orforglipron for obesity. With an anticipated decision as early as Q2 2026, this therapy could drive weight loss (11.2% weight loss seen in Phase 3 study) in a convenient, once-daily pill, broadening access and intensifying competition in the rapidly expanding obesity treatment market.  

4. Regeneron’s DB-OTO for congenital hearing loss (otoferlin gene variants). With an anticipated decision in the first half of 2026, this product could become the first gene therapy to restore near‑normal, durable hearing by correcting the underlying otoferlin gene defect, offering a one‑time, disease‑modifying alternative to lifelong cochlear implants for children born with congenital deafness.  

5. Sanofi’s teplizumab-mzmv (Tzield) for type 1 diabetes. With a potential decision in first half of 2026, approval for this monoclonal antibody could expand to delay the progression of stage 3 type 1 diabetes in ages 8 years and older in individuals recently diagnosed with stage 3 type 1 diabetes by offering the first disease-modifying therapy to delay the progression of type 1 diabetes by preserving pancreatic beta cell function, shifting care from lifelong insulin management toward earlier immune intervention in a disease with decades of unmet need.These first five approvals are part of the Commissioner’s National Priority Voucher (CNPV) pilot, a new initiative introduced by Dr. Makary, which awarded vouchers to several drug companies and aims to shorten the FDA’s review times from 10–12 months to just 1–2 months following filing of a complete drug or biologic application, while maintaining FDA’s rigorous safety and efficacy standards.  

Of note, Disc Medicine’s bitopertin for porphyria, which received the voucher program, was recently rejected by the FDA, marking the first known complete response letter under the CNPV program.  

The remaining six investigational drugs include options for both chronic and rare conditions.  

1. Rocket Pharmaceutical’s intravenous marnetegragene autotemcel (Kresaldi) for leukocyte adhesion deficiency-I. With an anticipated decision on March 28, 2026, this therapy would offer a potentially curative one-time gene therapy for a serious rare pediatric genetic immune disorder, offering an alternative to allogenic hematopoietic stem cell transplant for a disease that is otherwise frequently fatal in childhood. 

2. Chiesi’s oral idebenone for Leber’s hereditary optic neuropathy. With an anticipated decision on Feb. 28, 2026, this treatment would be the first FDA-approved option for a rare inherited mitochondrial disorder that leads to vision loss, addressing a major unmet need. It works by targeting mitochondrial dysfunction to preserve or improve vision in a rapidly blinding disease.  

3. Ascendis Pharma’s navepegritide for achondroplasia. With an anticipated decision on February 28, 2026, this treatment would potentially improve growth and body proportionality in children with a type of achondroplasia, offering a new once-weekly option beyond daily injections.  

4. GSK’s linerixibat for cholangitis pruritus. With an anticipated decision on March 24, 2026, this treatment would be the first of its kind developed to treat intense itching due to a liver disease. 

5. Gilead’s bulevirtide for chronic hepatitis D virus. With an anticipated decision in the first half of 2026, this medicine would offer the first treatment option for a severe and even fatal viral infection affecting the liver that could progress to liver fibrosis and failure. 

6. AstraZeneca’s camizestrant for selective estrogen-receptor (ER) degrader (SERD) and complete ER antagonist breast cancer. With an anticipated decision in the first half of 2026, this next-generation oral therapy could potentially reshape first-line treatment for hormone receptor-positive, HER-2 negative advanced breast cancer with a certain mutation by overcoming estrogen-receptor-driven resistance before disease progression. 

The drug pipeline is fluid. To keep atop of the latest on the drug pipeline, subscribe to the Prime Post newsletter to receive the latest insights so you are well-sourced on the latest developments in the drug pipeline and what’s ahead.  

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