Your monthly synopsis of new drugs expected to hit the market
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 United States (U.S.) Food and Drug Administration (FDA). 

Drug pipeline for January 2025

January – March 2025: bentracimab 
Bentracimab is a monoclonal antibody reversal agent for the P2Y₁₂ platelet inhibitor ticagrelor (Brilinta). SERB Pharmaceuticals submitted bentracimab to the FDA for use in a hospital setting in patients receiving ticagrelor who require a nondeferrable surgery/invasive procedure or who are experiencing major bleeding. The FDA granted bentracimab Breakthrough Therapy designation and a Priority Review. Bentracimab was evaluated in the RESERVE-IT trial as an intravenous (IV) bolus administered over 10 minutes, immediately followed by an IV loading infusion over 4 hours and then an IV maintenance infusion over 12 hours. Published interim data from the trial revealed that bentracimab provided reversal of ticagrelor’s antiplatelet effects within 5 to 10 minutes after the start of the bentracimab infusion.¹ The reversal effects lasted for over 24 hours. If approved, bentracimab will be the first reversal agent for ticagrelor and will fulfill an unmet need in patients prescribed the antiplatelet agent who require urgent surgery or are experiencing major bleeding.  

Jan. 7, 2025: vanzacaftor/tezacaftor/deutivacaftor (vanza triple) 
Vertex is awaiting an FDA decision for their triple combination therapy for the treatment of cystic fibrosis (CF) in patients six years and older with at least one F508del mutation or another responsive mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. Vanza triple contains three CFTR modulators, including the CFTR correctors vanzacaftor and tezacaftor and the CFTR potentiator deutivacaftor. In the SKYLINE 102 and SKYLINE 103 trials, in patients 12 years and older, once-daily vanza triple was noninferior in improving lung function (as measured by change in percent predicted forced expiration volume in on 1 second [ppFEV₁]) and superior in restoring CFTR function (as measured by sweat chloride levels) compared to twice-daily elexacaftor/tezacaftor/ivacaftor (Trikafta).² The single-arm RIDGELINE safety study revealed a similar safety profile for vanza triple in patients six to 11 years of age compared to those 12 years and older. Notably, while vanza triple did not result in an improvement in ppFEV₁ in the younger age group compared to baseline on Trikafta, it did improve CFTR function. The FDA granted vanza triple Fast Track and Orphan Drug designations and a Priority Review. If vanza triple is approved, Vertex expects that most patients on twice-daily oral Trikafta, also by Vertex, who are six years of age and older may switch to once-daily oral vanza triple.  

For more information, see the vanzacaftor/tezacaftor/deutivacaftor Deep Dive in the October 2024 edition of Prime Therapeutics’ Quarterly Drug Pipeline Quarterly Drug Pipeline: October 2024 - Prime Therapeutics - Portal 

Jan. 6, 2025: trastuzumab (TX05) 
Tanvex is seeking FDA approval of TX05, a trastuzumab biosimilar candidate to Genentech’s Herceptin. Herceptin is a human HER2/neu receptor antagonist indicated for the treatment of HER2-overexpressing breast cancer and gastric cancer. If approved, TX05 will be the seventh approved biosimilar to Herceptin.

Jan. 7. 2025: remestemcel-L (Ryoncil) 
Mesoblast is awaiting FDA decision for Ryoncil for the treatment of children with steroid-refractory, acute graft versus host disease (SR-aGVHD). This is the third review of the agent after the FDA issued Complete Response Letters (CRLs) in October 2020 and August 2023, requiring additional clinical data in each. Ryoncil is an off-the-shelf treatment that contains culture expanded mesenchymal stromal cells obtained from unrelated donor bone marrow. The cellular therapy is designed to inhibit the inflammatory processes that are associated with SR-aGVHD by inhibiting activation and proliferation of effector T cells, down-regulating the production of pro-inflammatory cytokines, and enabling recruitment of anti-inflammatory cells to affected tissue. Ryoncil was given Fast Track and Orphan Drug designations and a Priority Review by the FDA. It was evaluated in a single-arm, phase 3 trial (NCT02336230) as first-line treatment in children with SR-aGVHD following allogeneic hematopoietic stem cell transplant (HSCT).³ In the trial, Ryoncil was administered as an IV infusion twice per week for four consecutive weeks. The trial met its primary endpoint with an overall response rate (ORR) at day 28 of 70.4% compared to 45% for a prespecified control (p=0.0003). Ryoncil led to an overall survival (OS) of 74.1% at day 100 and 68.5% at day 180. Moreover, an observational cohort survival study of phase 3 data performed by the Center for International Blood and Marrow Transplant Research (CIBMTR) reported Ryoncil led to an OS of 67% at six months, 63% at one year, 51% at two years, and 49% at four years in children with SR-aGVHD.⁴ SR-aGVHD is associated with mortality as high as 90%. If approved, Ryoncil will be the first allogeneic off-the-shelf cellular therapy to be approved in the U.S. and the first treatment approved for SR-aGVHD in children less than 12 years of age. 

Jan. 10, 2025: insulin aspart (AMP004)  
Amphastar is awaiting FDA approval of AMP004, an insulin aspart biosimilar candidate to Novo Nordisk’s Novolog. Novolog is a rapid acting human insulin analog indicated to improve glycemic control in adults and pediatric patients with diabetes mellitus. If approved, AMP004 will be the first biosimilar to Novolog. 

Jan. 15, 2025: tabelecleucel (tab-cel)
Tabelecleucel (tab-cel) is an allogeneic, Epstein-Barr virus (EBV)-specific T-cell immunotherapy that eliminates EBV infected cells in a targeted manner. Pierre Fabre and Atara have submitted the cellular therapy to the FDA to be used as monotherapy for the treatment of EBV-positive (EBV+) post-transplant lymphoproliferative disease (PTLD) in patients 2 years of age and older who have received at least one prior therapy. The reported incidence of PTLD ranges from 1% to 30% depending on the transplanted organ. Tab-cel is an off-the-shelf therapy that can be individualization based on certain patient disease criteria. The FDA granted tab-cel Breakthrough Therapy and Fast Track designations and a Priority Review. If approved, tab-cel will be the first treatment specifically indicated for EBV+ PTLD and will likely be used as second-line treatment following rituximab. The open-label ALLELE trial evaluated tab-cel in patients with refractory or relapsed EBV+ PTLD following a HSCT or solid organ transplant (SOT).⁵ The study demonstrated an ORR of 51.2%, median duration of response (DOR) of 23 months, and median OS of 18.4 months. No episodes of cytokine release syndrome, immune effector cell-associated neurotoxicity syndrome or transplant rejection related to tab-cel were reported. In the clinical trial, tab-cel was administered IV over 5 to 10 minutes on days 1, 8, and 15 in 35-day cycles and the median number of treatment courses required was two cycles in the SOT recipients and three cycles in the HSCT recipients. On Oct. 31, 2024, the Institute for Clinical and Economic Review (ICER) published an evidence report on the treatment of EBV+ PTLD. Although data is limited, based on positive safety and efficacy, the panel has high certainty that tab-cel could provide a substantial net health benefit (“A”) compared with usual care.⁶

For more information, see the tabelecleucel Deep Dive in the October 2024 edition of Prime Therapeutics’ Quarterly Drug Pipeline Quarterly Drug Pipeline: October 2024 - Prime Therapeutics - Portal  

Jan. 28, 2025: tocilizumab (CT-P47) 
Celltrion is seeking FDA approval of CT-P47, a tocilizumab biosimilar candidate to Genentech’s Actemra. Actemra is an interleukin-6 (IL-6) receptor antagonist that is indicated in select patients with rheumatoid arthritis, giant cell arteritis, interstitial lung disease, juvenile idiopathic arthritis, and cytokine release syndrome, as well as COVID-19 in the hospital setting. If approved, CT-P47 will be the third approved biosimilar to Actemra. 

Jan. 29, 2025: elamipretide 
Stealth Biotherapeutics submitted elamipretide for the treatment of Barth syndrome, an ultra-rare metabolic and neuromuscular genetic disorder that primarily affects men. Characteristic manifestations include cardiomyopathy, with onset usually before five years of age, as well as neutropenia, skeletal myopathy and growth delay. Elamipretide is a first-in-class mitochondria-targeted agent. It was evaluated in a single-site, blinded, placebo-controlled, crossover phase 2/3 TAZPOWER trial in 12 ambulatory males 12 years of age and older with confirmed Barth syndrome.⁷ Elamipretide was administered as a 40 mg subcutaneous (SC) injection once daily. While elamipretide did not result in significant improvements in the 6-minute walk test (6MWT; primary endpoint) or in fatigue scores or muscle strength (secondary endpoints) compared with placebo at the 12 weeks, increasing and persistent improvements that reached statistical significance were observed across these domains during the open-label extension period. After 24 total weeks of drug exposure, the mean 6MWT improved by 16% improvement (60.5 meters; p=0.02), fatigue scores decreased by 19% (p=0.03), and there was a 30% improvement in hand strength (measured by handheld dynamometry; p=0.003). Elamipretide was generally well-tolerated. Elamipretide has Orphan Drug, Fast Track and Rare Pediatric Disease designations from the FDA and was granted a Priority Review. In addition, the FDA’s Cardiovascular and Renal Drugs Advisory Committee (CRDAC) voted 10 to 6 in favor of the effectiveness of elamipretide for treating Barth syndrome. If approved, elamipretide will be the first treatment available for Barth syndrome. 

Jan. 30, 2025: suzetrigine 
Vertex is awaiting FDA approval of suzetrigine, a non-opioid, oral, selective NaV1.8 pain signal inhibitor, for the treatment of moderate to severe acute pain. Suzetrigine was evaluated in surgical (post abdominoplasty or bunionectomy) and non-surgical settings. Interim data in post-surgical patients demonstrated that suzetrigine led to a significant improvement in pain intensity compared to placebo, as measured by pain intensity difference from 0 to 48 hours (SPID48), and to a faster onset of a meaningful relief in pain (≥ 2-point reduction on the Numeric Pain Rating Scale) compared to placebo.⁸ However, suzetrigine was not superior to hydrocodone bitartrate/acetaminophen for relief of post-surgical pain. A single-arm trial that evaluated suzetrigine in a broad range of surgical and non-surgical adult patients reported  83.2% of patients described suzetrigine’ s effectiveness in treating pain as good, very good or excellent based on patient global assessment (PGA) scores. In the clinical trials, suzetrigine was administered orally with an initial dose of 100 mg followed by 50 mg every 12 hours for up to 14 days. The FDA granted suzetrigine Breakthrough Therapy and Fast Track designations as well as a Priority Review. If approved, suzetrigine will provide the first new mechanism of action for the short-term (up to 14 days) treatment of acute pain in about 20 years. 

For more information, see the suzetrigine Deep Dive in the October 2024 edition of Prime Therapeutics’ Quarterly Drug Pipeline Quarterly Drug Pipeline: October 2024 - Prime Therapeutics - Portal 

Jan. 31, 2025: apomorphine infusion device (SPN-830) 
Supernus submitted SPN-830, non-ergoline dopamine agonist, to the FDA for the continuous treatment of motor fluctuations (off episodes) in Parkinson’s disease. This is the fourth submission to the FDA for the continuous subcutaneous infusion pump, after the agency refused to file the application in November 2020, citing an incomplete application, and issued CRLs in October 2022 and April 2024 requiring additional information and analysis related to the infusion device and drug product. Published data from the European double-blind, placebo-controlled, phase 3 TOLEDO trial reported apomorphine infusion significantly reduced off time compared with placebo (difference, -1.89 hours per day, p=0.0025) in patients with motor fluctuations associated with Parkinson’s disease not adequately controlled by available treatment.⁹ If approved, SPN-830 will be the second apomorphine formulation available in the U.S., following the approval of Apokyn for the acute intermittent treatment off episodes in patients with advanced Parkinson’s disease. Apokyn may be self- or caregiver-administered via SC injection using a multiple-dose pen injector and can be dosed up to five times a day, with doses at least two hours apart. 

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