This year, the FDA is expected to approve several new therapies that highlight the increasing complexity of pharmaceutical manufacturing.
From biologics to treatments for rare diseases, these drugs will bring unique production challenges that require advanced technologies and precise processes. Here’s a look at six anticipated approvals in 2025.
AXS-07
Manufacturer: Axsome Therapeutics
Indication: Acute treatment of migraine
AXS-07 is an oral agent combining meloxicam, a COX-2 preferential nonsteroidal anti-inflammatory drug, and rizatriptan, a 5-HT1B/1D agonist.
In May 2022, the company received a Complete Response Letter regarding its NDA submission for AXS-07, highlighting concerns related to chemistry, manufacturing, and controls. The agency did not request any additional clinical trials at that time.
In September 2024, the FDA accepted Axsome Therapeutics’ resubmitted new drug application (NDA) for AXS-07 as a treatment for acute migraine. The agency assigned a Prescription Drug User Fee Act (PDUFA) decision date of January 31, 2025, for the oral, rapidly absorbed, multi-mechanistic investigational medicine.
Suzetrigine
Manufacturer: Vertex Pharmaceuticals
Indication: Moderate to severe acute pain
Suzetrigine is an oral, selective inhibitor of NaV1.8, a voltage-gated sodium channel that plays a critical role in pain signaling in the peripheral nervous system. For neuropathic pain, specifically pain associated with diabetic peripheral neuropathy (DPN), Vertex has also successfully completed an end-of-Phase 2 meeting with the FDA.
Following positive Phase 2 results, the company was preparing to launch a Phase 3 study in the latter half of 2024. Suzetrigine also received the FDA’s Breakthrough Therapy designation for its application in DPN treatment.
Datopotamab Deruxtecan (Dato-DXd)
Manufacturers: AstraZeneca and Daiichi Sankyo
Indication: Unresectable or metastatic hormone receptor-positive, HER2-negative breast cancer
Developed with Daiichi Sankyo, Dato-DXd is a TROP2-directed DXd ADC, designed to target a protein called TROP2 found in cancer cells. Dato-DXd is made up of an antibody that recognizes TROP2 and a medication that can kill cancer cells attached to it.
The companies have several ongoing trials for the ADC. In their TROPION-Breast02 trial, the drug is being compared to chemotherapy in patients with previously untreated locally recurrent inoperable or metastatic triple negative breast cancer who are ineligible for anti-PDL1 therapy. Meanwhile, in TROPION-Breast03, Dato-DXd is being evaluated in combination with or without durvalumab, as compared to the first in line therapies for patients with stage I-III TNBC who still have residual disease after neoadjuvant therapy.
Tab-cel
Manufacturer: Atara Biotherapeutics
Indication: Epstein-Barr virus (EBV)–positive post-transplant lymphoproliferative disease
Tab-cel is an allogeneic, EBV-specific T-cell immunotherapy for the treatment of patients with EBV-positive post-transplant lymphoproliferative disease following at least one prior therapy.
In December 2023, Atara announced the expansion of its global partnership with Pierre Fabre Laboratories to include the U.S. and additional global commercial markets for tab-cel, building on their initial agreement covering Europe, the Middle East, Africa, and select emerging markets. Following the acceptance of the tab-cel BLA, Atara will receive a $20 million milestone payment from Pierre Fabre, with the possibility of an additional $60 million contingent on FDA approval.
Pierre Fabre is also covering Atara’s expected global development costs for tab-cel through the BLA transfer and purchasing inventory through the manufacturing transfer date. Additionally, Atara is eligible for sales milestones and double-digit tiered royalties on net sales of tab-cel in the newly included markets.
Vanzacaftor/Tezacaftor/Deutivacaftor (Vanza Triple)
Manufacturer: Vertex Pharmaceuticals
Indication: Cystic fibrosis
The investigational combination therapy vanzacaftor/tezacaftor/deutivacaftor targets the underlying cause of cystic fibrosis by improving CFTR protein function. Vanzacaftor and tezacaftor enhance the processing and trafficking of CFTR protein to the cell surface, while deutivacaftor increases the protein’s channel open probability to improve salt and water flow across cell membranes.
The triple therapy received Fast Track and Orphan Drug Designations from the FDA and will carry a lower single-digit royalty obligation compared to Vertex’s current cystic fibrosis treatments.
CUTX-101 (Copper Histidinate)
Manufacturer: Cyprium Therapeutics
Indication: Menkes disease
CUTX-101, developed by Cyprium Therapeutics, is a promising therapy for Menkes disease, a rare and life-threatening genetic disorder that affects copper absorption and metabolism. The condition leads to severe developmental delays, connective tissue issues, and neurological decline.
The therapy has received multiple designations from the FDA, including Breakthrough Therapy, Fast Track, Rare Pediatric Disease, and Orphan Drug, as well as Orphan Designation from the EMA. The FDA set a PDUFA target action date of June 30, 2025.
In December 2023, Sentynl took over the development and commercialization of CUTX-101 from Cyprium Therapeutics, a Fortress subsidiary.
