Insights+: The US FDA New Drug Approvals in March 2024

Shots:

PharmaShots has compiled a list of US FDA-approved drugs in the month of March 2024
The US FDA approved a total of 14 drugs including 12 new molecular entities and 2 biologics leading to the treatments for patients and advances in the healthcare industry
The major highlighted drug was BeiGene’s Tevimbra for treating Advanced or Metastatic Esophageal Squamous Cell Carcinoma

1.Formosa Pharmaceuticals Receives the US FDA’s Approval for Clobetasol Propionate to Treat Post-Operative Inflammation and Pain Following Ocular Surgery

Active Ingredient: Clobetasol Propionate

Approved: Mar 04, 2024

Company: Formosa Pharmaceuticals              

Disease: Post-Operative Inflammation and Pain Following Ocular Surgery

The US FDA has approved Formosa’s clobetasol propionate ophthalmic suspension 0.05% (APP13007) to treat post-operative inflammation & pain following ocular surgery
Sosei Heptares will receive a payment of $2.5M from Formosa on achieving the milestone as per the agreement signed b/w them
APP13007 was initially developed by Activus Pharma (subsidiary of Sosei) using its Activus Pure Nanoparticle Technology. It was divested to Formosa in Aug 2017

2. The US FDA Grants Approval to BeiGene’s Tevimbra to Treat Advanced or Metastatic Esophageal Squamous Cell Carcinoma

Active Ingredient: Tislelizumab-jsgr

Approved: Mar 13, 2024

Company: BeiGene          

Disease: Esophageal Squamous Cell Carcinoma

BeiGene’s Tevimbra has received the US FDA’s approval as a monotx. to treat unresectable or metastatic esophageal squamous cell carcinoma (ESCC) after previous systemic chemotherapy (CT) excl. PD-(L)1 inhibitor. The US availability is anticipated in H2’24
The approval was supported by the P-III (RATIONALE 302) study assessing the safety & efficacy of Tevimbra vs CT for the 2L treatment of unresectable, locally advanced or metastatic ESCC patients (n=512) across the EU, Asia & North America
The study met the 1EP among the ITT population demonstrating mOS of 8.6mos. vs 6.3mos. with a favorable safety outcome

3. Madrigal Pharmaceuticals’ Rezdiffra (resmetirom) Receives the US FDA’s Approval to Treat NASH with Liver Fibrosis

Active Ingredient: Resmetirom

Approved: Mar 14, 2024

Company: Madrigal Pharmaceuticals         

Disease: NASH with Liver Fibrosis

The US FDA has granted accelerated approval to Rezdiffra for treating noncirrhotic NASH with moderate to advanced liver fibrosis (F2 to F3) supported by the P-III (MAESTRO-NASH) study assessing its safety & efficacy at 100mg & 80mg dosing vs PBO in NASH patients (n=1,759)
The results after 52wks. showed improved NASH resolution (incl. a ≥2 points reduction in the NAFLD activity score) without worsening fibrosis & fibrosis improvement by at least one stage without worsening NAFLD activity score
The MAESTRO-NASH study continues for full approval while a 2nd study is underway to assess the progression to liver decompensation events in well-compensated NASH cirrhosis patients treated with Rezdiffra vs PBO

4. Idorsia’s Tryvio Receives the US FDA’s Approval for the Treatment of Hypertension

Active Ingredient: Aprocitentan

Approved: Mar 20, 2024

Company: Idorsia

Disease: Hypertension

The US FDA has approved Tryvio (12.5mg, oral, QD, with/without food) in addition with antihypertensive drugs to treat hypertensive adults not controlled on other drugs
The P-III (PRECISION) trial assessed the efficacy of Tryvio for hypertensive adults with SBP ≥140 mmHg receiving at least 3 antihypertensive therapies with the 1EP as change in SiSBP at wk.4 & 2EP as change in SiSBP from wk.36 to wk.40
In the study post 4wks. PBO run-in duration, patients (n=730) in part 1 were administered Tryvio (12.5mg, 25mg, or PBO, QD) for 4wks., all patients were then administered Tryvio (25mg, QD) for 32wks. in part 2 & post 32wks. patients were re-randomized to receive Tryvio (25mg) or PBO for a 12wk. withdrawal period in part 3

5. Italfarmaco’s Duvyzat Obtains the US FDA’s Approval to Treat Duchenne Muscular Dystrophy

Active Ingredient: Givinostat

Approved: Mar 22, 2024

Company: Italfarmaco       

Disease: Duchenne Muscular Dystrophy

The US FDA has granted approval to Italfarmaco’s Duvyzat (givinostat), histone deacetylase (HDAC) inhibitor, to treat Duchenne muscular dystrophy (DMD) patients (6yrs. or above)
The approval was supported by the P-III (EPIDYS) study assessing the safety & efficacy of Duvyzat (BID) vs PBO in DMD ambulant male patients (n=179, 6yrs. or above)
The trial met the 1EP showing a meaningful difference in time to complete the four-stair climb evaluation. Favorable outcomes were also observed across 2EPs such as NSAA & fat infiltration assessment through MRI. The findings were published in The Lancet Neurology

6. Johnson & Johnson’s Opsynvi (macitentan and tadalafil) Receives the US FDA’s Approval for the Treatment of Pulmonary Arterial Hypertension

Active Ingredient: Macitentan and Tadalafil

Approved: Mar 26, 2024

Company: Johnson & Johnson     

Disease: Pulmonary Arterial Hypertension

The approval was based on the results from the P-III (A DUE) clinical trial evaluating the safety & efficacy of Opsynvi vs macitentan/tadalafil monotx. in patients with PAH (WHO FC II or III). The 1EP was a change from baseline in Pulmonary Vascular Resistance (PVR) at 16wks.
The study depicted a greater reduction in PVR at 16wks. with Opsynvi vs macitentan/tadalafil monotx. The trial thereby met its co-primary endpoints by depicting a significant pulmonary hemodynamic improvement
Opsynvi is a combination of macitentan (endothelin receptor antagonist) & tadalafil (phosphodiesterase 5 (PDE5) inhibitor)

7. Merck’s Winrevair (sotatercept-csrk) Receives the US FDA’s Approval for the Treatment of Pulmonary Arterial Hypertension (PAH)

Active Ingredient: Sotatercept-csrk

Approved: Mar 27, 2024

Company: Merck 

Disease: Pulmonary Arterial Hypertension

The approval was based on the results from the P-III (STELLAR) trial evaluating Winrevair (0.7mg/kg) vs PBO + stable background therapy (SC, Q3W) in patients (n=163 vs 160) with PAH. The 1EP was change from baseline at wk.24 in 6MWD
The results depicted a 41m median increase in 6MWD with Winrevair vs PBO along with an improvement in FC at wk.24 in 29% vs 14% of patients, an 84% reduction in the occurrence of death or PAH clinical worsening events, and improvement in PVR & NT-proBNP levels from baseline with Winrevair vs PBO
Winrevair is an activin signaling inhibitor therapy that improves the balance between pro-proliferative & anti-proliferative signaling to modulate vascular proliferation. The US FDA earlier granted BTD to Winrevair

8. Akebia’s Vafseo (Vadadustat) Tablets Gains the US FDA’s Approval to Treat Anemia Due to CKD

Active Ingredient: Vadadustat

Approved: Mar 27, 2024

Company: Akebia

Disease: Anemia Due to CKD

The US FDA has approved the company’s Vafseo (vadadustat) tablets as a treatment of anemia associated with chronic kidney disease (CKD) in adults on dialysis for at least 3mos. The company, in collaboration with CSL Vifor, will commercialize Vafseo across the US
The approval was based on the safety and efficacy results from INNO2VATE trial and an evaluation of safety outcomes after marketing in Japan. The results were published in the New England Journal of Medicine
Vafseo (oral, QD) is a HIF-PH inhibitor that triggers the body's response to hypoxia by promoting the production of erythropoietin to address anemia

Note: According to the FDA's March 2024 approval list, Talzenna (Mar 07, 2024), Cabazitaxel (Mar 14, 2024), Edurant PED (Mar 15, 2024), Efavirenz, Emtricitabine & Tenofovir Alafenamide (Mar 22, 2024), Opsynvi (Mar 22, 2024) and Risvan (Mar 29, 2024) were also approved; however, no PR was available