January and March 2026 drug pipeline updates span Phase 3, Fast Track and Priority Review
January and March 2026 drug pipeline updates included Phase 3 results, Fast Track designations and Priority Review decisions across multiple disease areas. Mavacamten, teplizumab-mzwv, anifrolumab, AXS-05, gedatolisib and AFTX-201 were among the programs with notable milestones.
Notable updates occurred in January 2026 and March 2026 for investigational products in development. Clinical and regulatory milestones included Phase 3 results, Fast Track designation, Breakthrough Therapy designation, Priority Review, and applications accepted for review across cardiovascular, immune, neurologic, oncology, endocrine, and other disease areas.
In cardiovascular disease, Mavacamten posted Phase 3 results in symptomatic obstructive hypertrophic cardiomyopathy in adolescents, while AFTX-201 received Fast Track designation for treatment of BAG3-associated dilated cardiomyopathy. In endocrine disorders, Teplizumab-mzwv had an sBLA accepted for Priority Review to delay stage 3 type 1 diabetes in pediatric patients aged 1 to 7 years with stage 2 type 1 diabetes, GZR4 reported Phase 3 results in adults with type 2 diabetes mellitus, and Retatrutide reported Phase 3 results in adults with type 2 diabetes and inadequate glycemic control.
Immune and inflammatory disease updates included Anifrolumab with Phase 3 results in systemic lupus erythematosus, Atacicept with a BLA accepted for Priority Review in immunoglobulin A nephropathy, Ianalumab with Breakthrough Therapy designation in Sjögren disease, Litifilimab with Breakthrough Therapy designation in cutaneous lupus erythematosus, and Obexelimab with Phase 3 results in IgG4-related disease. In March, Brepocitinib received Priority Review for adults with dermatomyositis, BW-20805 received Fast Track designation for hereditary angioedema, and Nipocalimab received Fast Track designation for active systemic lupus erythematosus.
Neurologic updates included AXS-05 with Priority Review in Alzheimer disease agitation, Alixorexton with Breakthrough Therapy designation in narcolepsy type 1, Dexamethasone sodium phosphate in a Phase 3 trial for ataxia-telangiectasia, Levacetylleucine with Phase 3 results in ataxia-telangiectasia, and Lecanemab-irmb with an sBLA accepted for review for a subcutaneous starting dose in Alzheimer disease with mild cognitive impairment or mild dementia. In March, Fenebrutinib reported Phase 3 results in relapsing multiple sclerosis, Relutrigine received Priority Review in SCN2A and SCN8A developmental and epileptic encephalopathies, and Zilganersen received Priority Review in Alexander disease.
Oncology milestones included Gedatolisib with an NDA accepted for review in HR+/HER2-, PIK3CA wild-type advanced breast cancer, Ivonescimab with a BLA accepted for review in EGFR-mutated locally advanced or metastatic non-squamous non-small cell lung cancer, Relacorilant with Phase 3 results in platinum-resistant ovarian cancer, Sofetabart mipitecan with Breakthrough Therapy designation in platinum-resistant epithelial ovarian, fallopian tube, or primary peritoneal cancer, and BNT113 with Fast Track designation in HPV16+ head and neck squamous cell carcinoma. March updates added Bezuclastinib with an NDA accepted for review in nonadvanced systemic mastocytosis, Fam-trastuzumab deruxtecan-nxki with Priority Review in HER2-positive breast cancer with residual invasive disease after neoadjuvant therapy, Mezigdomide, Selinexor, and Talazoparib with Phase 3 results, and TRI-611 with Fast Track designation in ALK+ non-small cell lung cancer.
Other January updates included Amlitelimab with Phase 3 results in moderate to severe atopic dermatitis, Ropeginterferon alfa-2b-njft with an sBLA accepted for review in essential thrombocythemia, QTX-2101 with Fast Track designation in acute promyelocytic leukemia, Bepirovirsen with Phase 3 results in chronic hepatitis B, Efgartigimod alfa-fcab with Priority Review in AChR-Ab seronegative generalized myasthenia gravis, Ixekizumab and tirzepatide with Phase 3 results in active psoriatic arthritis in patients with obesity or overweight, Centanafadine with Priority Review in attention-deficit hyperactivity disorder, and Sparsentan with a review period extended in focal segmental glomerulosclerosis.
Other March updates included Lebrikizumab-libkz with Phase 3 results in moderate to severe atopic dermatitis in pediatric patients as young as 6 months, Rusfertide with Priority Review in adults with polycythemia vera, Sutacimig with Breakthrough Therapy designation for prevention of bleeding episodes in patients with Glanzmann thrombasthenia, PF-07307405 with Phase 3 results for prevention of Lyme disease, DTX301 with Phase 3 results in late-onset ornithine transcarbamylase deficiency, and Elegrobart with Phase 3 results in active thyroid eye disease.