▎WuXi AppTec Content Team Report

According to public information, this week (August 12th ~ August 17th), at least six new drugs received approval from the U.S. FDA for marketing. After organizing the information, these new drugs include several antibody therapies, oral sustained-release capsules, long-acting prodrug medications, etc., which will be used to treat diseases such as lung cancer, Parkinson's disease, hypoparathyroidism, prurigo nodularis, and primary biliary cholangitis.

AstraZeneca:Imfinzi（durvalumab）

Approved Indications:Non-Small Cell Lung Cancer

On August 17, AstraZeneca announced its重磅PD-L1 targeted antibodyImfinzi (durvalumab) in combination with chemotherapy receives FDA approval for the treatment of resectable, early-stage (IIA-IIIB) non-small cell lung cancer (NSCLC) adult patients whose tumors have no known epidermal growth factor receptor (EGFR) mutation or anaplastic lymphoma kinase (ALK) Rearrangement.In this regimen, patients receive Imfinzi combined with neoadjuvant chemotherapy before surgery and Imfinzi monotherapy as adjuvant treatment after surgery. Results from a previous Phase 3 trial showed,Patients receiving Imfinzi-based regimen treatmentThe pathological complete response (pCR) rate was four times that of the control group.。Imfinzi is a humanized monoclonal antibody. By binding to PD-L1, Imfinzi blocks the interaction between PD-L1 and PD-1 as well as CD80 proteins, thereby inhibiting the tumor's immune escape mechanism.

Amneal Pharmaceuticals：Crexont (Carbidopa and Levodopa) Extended-Release Capsules

Approved Indications:Parkinson's Disease (PD)

Recently, Amneal Pharmaceuticals announced that the FDA has approved Crexont (carbidopa and levodopa) extended-release capsules for marketing to treat Parkinson's disease.Crexont is an innovative oral formulation,It contains immediate-release granules of carbidopa and levodopa for quick onset, as well as extended-release granules of levodopa to provide prolonged efficacy.Is expected to provide a better treatment experience for patients with Parkinson's disease.

Incyte/Syndax Pharmaceuticals：Niktimvo（axatilimab）

Approved Indications:Chronic Graft-Versus-Host Disease（cGVHD）

On August 15, the FDA announcedApproval of Incyte Corporation and Syndax Pharmaceuticals' jointly developed antibody therapy Niktimvo (axatilimab) for the treatment of chronic graft-versus-host disease.(cGVHD) adult and pediatric patients weighing over 40 kilograms who have previously received at least two systemic treatments.

This approval is based on the randomized, open-label, multicenter clinical trial named AGAVE-201. The trial evaluated the efficacy of three doses of Niktimvo in treating patients with relapsed or refractory cGVHD.The primary efficacy endpoint was the overall response rate (ORR),Among the 79 patients who received the recommended dose, the ORR of Niktimvo reached 75%.Among the patients who achieved remission, 60% survived without death or receiving new systemic treatment for at least 12 months.Niktimvo is a monoclonal antibody targeting colony-stimulating factor-1 receptor (CSF-1R).In September 2021, Syndax entered into a global exclusive development and commercialization licensing agreement with Incyte for the joint development of Niktimvo.

Gilead Sciences:Livdelzi（seladelpar）

Approved Indications:Primary Biliary Cholangitis (PBC)

On August 15, Gilead Sciences announced that the FDA granted accelerated approval to Livdelzi (seladelpar) for use in combination with ursodeoxycholic acid (UDCA) to treat adult patients with primary biliary cholangitis (PBC) who have an inadequate response to UDCA, or as monotherapy for patients intolerant to UDCA. It is not recommended for patients with or developing decompensated cirrhosis.

LivdelziAn orally administered potent and selective peroxisome proliferator-activated receptor δ (PPAR δ) agonist.PPARδ is expressed in various cell types in the liver and regulates multiple genes involved in bile acid synthesis, inflammation, and fibrosis processes. The drug received Breakthrough Therapy Designation from the FDA in February 2019 for the treatment of PBC. Originally developed by CymaBay, Gilead Sciences reached a deal with the company in February 2024 that could reach up to $4.3 billion.Acquisition Agreement, and thereby acquire this therapy.

This accelerated approval is mainly based on data from the pivotal placebo-controlled Phase 3 RESPONSE study. In this study,62% of subjects receiving once-daily oral Livdelzi achieved the primary endpoint of combined biochemical response at Month 12, compared to only 20% in the placebo group.After treatment with Livdelzi, 25% of patients had normalized ALP values at the 12th month, whereas no such change occurred in the placebo group.ALP is a cholestatic marker that can predict the risk of liver transplantation and death.

Galderma：Nemluvio（nemolizumab）

Approved Indications:Adult Prurigo Nodularis

On August 14, Galderma announced that the FDA had approved its interleukin-31 (IL-31) receptor-targeted monoclonal antibody Nemluvio (nemolizumab) for the treatment of adult patients with prurigo nodularis. Results from previous Phase 3 trials showed,As early as week 4, a rapid reduction in pruritus was observed in patients with prurigo nodularis receiving Nemluvio treatment.According to the press release,Nemluvio is the first monoclonal antibody to receive FDA approval for inhibiting IL-31 signaling.

Nemluvio is a "first-in-class" monoclonal antibody that blocks the IL-31 signaling pathway by binding to the IL-31 receptor α.Nemluvio was initially developed by Chugai Pharmaceutical Co., Ltd.This approval is mainly based on the positive results of the Phase 3 clinical project OLYMPIA.The Phase 3 clinical trials OLYMPIA 1 and OLYMPIA 2 evaluated the efficacy and safety of Nemluvio administered subcutaneously every four weeks in more than 500 patients with prurigo nodularis.OLYMPIA 1 and 2 trials both met the primary endpoint, andAchieved all other key secondary endpoints.

Ascendis Pharma：Yorvipath（palopegteriparatide）

Approved Indications:Adult Hypoparathyroidism

On August 13, Ascendis Pharma announced that the FDA approved Yorvipath (palopegteriparatide) for the treatment of adult hypoparathyroidism.According to the press release, Yorvipath isFirst FDA-ApprovedA drug for the treatment of adult patients with hypoparathyroidism.

Yorvipath (formerly known as TransCon PTH) is a once-daily long-acting prodrug of parathyroid hormone.It aims toParathyroid HormoneRestoration to physiological levels within 24 hours to address both the short-term symptoms and long-term complications of this condition. Phase 3The test results showed,Among patients treated with Yorvipath, 78.7% achieved independence from conventional therapy while maintaining serum calcium levels within the normal range (8.3–10.6 mg/dL), compared to only 4.8% in the placebo group.Moreover, both physical and cognitive symptoms detected by the Hypoparathyroidism Patient Experience Scale (HPES) were significantly reduced in a statistically significant manner.

[1] Official websites of various companies and publicly available information