NDA

LHON updates: Gene therapy progress; idebenone receives US CRL

Thu, 02 Apr 2026 12:00:00 -0400

Chiesi Group’s idebenone faced a regulatory setback last month after the U.S. FDA issued a complete response letter (CRL) to the company’s NDA for Leber hereditary optic neuropathy (LHON), a rare inherited disorder that causes sudden vision loss.But a chance missed for Chiesi may be an opportunity for gene therapies, including Gensight Biologics SA’s lenadogene nolparvovec (Lumevoq; GS-010).

Kintor’s KX-826 meets phase III endpoints in alopecia

Tue, 24 Mar 2026 12:00:00 -0400

Kintor Pharmaceutical Ltd.’s topical androgen receptor antagonist, pyrilutamide (KX-826), met the primary endpoint in a pivotal phase III trial in male androgenetic alopecia, and the company will soon file an NDA with China’s National Medical Products Administration.

Kintor’s KX-826 meets phase III endpoints in alopecia

Thu, 19 Mar 2026 12:00:00 -0400

Takeda’s oral narcolepsy drug steps closer to FDA approval

Tue, 24 Feb 2026 11:00:00 -0500

The U.S. FDA accepted, with priority review, Takeda Pharmaceutical Co. Ltd.’s NDA submission for oveporexton (TAK-681), bringing the oral orexin receptor 2 agonist closer to clearance in narcolepsy type 1.

Takeda’s oral narcolepsy drug steps closer to FDA approval

Wed, 11 Feb 2026 12:00:00 -0500

Genentech plans NDA for multiple sclerosis pill with study win

Tue, 10 Feb 2026 12:00:00 -0500

Genentech Inc. is covering more bases in multiple sclerosis, with its latest swing on fenebrutinib hitting a rare phase III noninferiority win against Ocrevus (ocrelizumab) in primary progressive multiple sclerosis.

Hair loss drugmaker Veradermics floats upsized $256M US IPO

Wed, 04 Feb 2026 12:00:00 -0500

Veradermics Inc. raised $256.3 million from an IPO on the New York Stock Exchange Feb. 4. The proceeds will fund three ongoing phase II/III clinical trials of VDPHL-01, its novel oral formulation of Rogaine (minoxidil) to treat pattern hair loss in men and women.

Elevar gets new CEO, files NDAs for lirafugratinib, rivoceranib

Tue, 03 Feb 2026 12:00:00 -0500

Elevar Therapeutics Inc. appointed Kim Dong-gun (DG) as CEO Jan. 29, as the company focuses on post-NDA strategies for lirafugratinib in bile duct cancer, and the twice-rejected rivoceranib-camrelizumab combination for liver cancer.

Elevar gets new CEO, files NDAs for lirafugratinib, rivoceranib

Fri, 30 Jan 2026 12:00:00 -0500

China accepts Hutchmed fanregratinib NDA on single phase II trial

Tue, 30 Dec 2025 10:00:00 -0500

Hutchmed (China) Ltd. has moved closer to establishing China’s first domestically developed FGFR-targeted therapy for intrahepatic cholangiocarcinoma, after the National Medical Products Administration (NMPA) accepted its NDA for fanregratinib (HMPL-453) and granted the drug priority review.

Immunome’s phase III results in desmoid tumors point to NDA

Mon, 15 Dec 2025 12:00:00 -0500

Positive top-line phase III data for Immunome Inc.’s oral, once daily treatment for progressing desmoid tumors have prompted the company to say it plans to submit an NDA to the U.S. FDA in the second quarter of next year. Varegacestat, an oral gamma secretase inhibitor, hit its primary endpoint by significantly improving progression-free survival compared to placebo. The small molecule produced a statistically significant and clinically meaningful improvement vs. placebo with its 84% reduction in the risk of disease progression or death.

14 psoriasis therapies projected to launch in China by 2027

Mon, 08 Dec 2025 12:00:00 -0500

Fourteen therapies to treat moderate to severe psoriasis are expected to enter the Chinese market in the next two years, according to Clarivate and BioWorld reports. Eleven of them are being developed by domestic biopharmaceutical firms.

A second win for Cogent pushes the company toward two NDAs

Mon, 10 Nov 2025 12:00:00 -0500

Cogent Biosciences Inc. is now lining up two NDA submissions for its tyrosine kinase inhibitor bezuclastinib in treating two forms of cancer. Cogent intends to submit an NDA for bezuclastinib, a tyrosine kinase inhibitor that targets and inhibits mutated KIT proteins, specifically KIT D816V, in the first half of 2026 to treat gastrointestinal stromal tumors. That will follow the company’s plans for an NDA submission for bezuclastinib in treating non-advanced systemic mastocytosis before the end of 2025.

Lepu’s MRG-003 cleared as China’s first EGFR ADC for advanced NPC

Tue, 04 Nov 2025 10:00:00 -0500

Lepu Biopharma Co. Ltd. said Oct. 30 it won Chinese approval of a novel antibody-drug conjugate (ADC), Meiyouheng (becotatug vedotin injection), making it China’s first epidermal growth factor receptor (EGFR)-directed ADC for advanced nasopharyngeal cancer (NPC).

Metis plans China NDA submission for orally dissolving PBA drug

Tue, 28 Oct 2025 11:00:00 -0400

Based on positive phase III study results, Metis Techbio is planning to file an NDA for its AI-derived orally disintegrating tablet drug candidate for pseudobulbar affect, MTS-004, in China next year.

Hanmi’s efpeglenatide hits phase III endpoints in obesity

Tue, 28 Oct 2025 11:00:00 -0400

Hanmi Pharmaceutical Co. Ltd. announced Oct. 27 that its glucagon-like peptide-1 receptor agonist, efpeglenatide (HM-11260C), met the co-primary endpoints in a phase III study of obese adults without diabetes.

Metis plans China NDA submission for orally dissolving PBA drug

Mon, 27 Oct 2025 11:00:00 -0400

Praxis soars on essential tremor phase III, preps for NDA

Thu, 16 Oct 2025 11:00:00 -0400

Success by nearly all measures with Praxis Precision Medicines Inc.’s essential tremor phase III candidate, ulixacaltamide, drove the company’s stock up significantly and provided solid data for an NDA filing planned for early 2026.

Taho submits US NDA for first apixaban oral dissolving film

Tue, 07 Oct 2025 11:00:00 -0400

Taho Pharmaceuticals Ltd. announced an NDA submission to the U.S. FDA for TAH-3311, developed as the world’s first oral dissolving film formulation of apixaban, marking a regulatory milestone for the Taipei-based biotech.

Taho submits US NDA for first apixaban oral dissolving film

Thu, 02 Oct 2025 11:00:00 -0400

Roivant, Priovant plan NDA on positive dermatomyositis data

Wed, 17 Sep 2025 11:00:00 -0400

With positive phase III results in hand, Roivant Sciences Ltd. and Priovant Therapeutics Inc. anticipate an NDA filing in the first half of 2026, seeking approval for brepocitinib, a dual inhibitor of tyrosine kinase 2 and JAK1, in dermatomyositis, a rare autoimmune disease with limited treatment options.

Saol Therapeutics hit with CRL for rare genetic disease

Tue, 09 Sep 2025 11:00:00 -0400

The U.S. FDA issued a complete response letter (CRL) for the NDA to privately held Saol Therapeutics Inc.’s rare disease treatment, sodium dichloroacetate (SL-1009), for pyruvate dehydrogenase complex deficiency. The inhibitor of pyruvate dehydrogenase kinases is the only drug in development for treating the rare genetic disorder, according to Cortellis. There are no FDA-approved treatments for the disease.

China clears mazdutide as first dual GCG/GLP-1RA for weight loss

Mon, 30 Jun 2025 11:00:00 -0400

Innovent Biologics Inc. announced June 27 that it gained National Medical Products Administration’s (NMPA) approval of mazdutide as a new weight loss therapy for obese or overweight patients in China. Mazdutide is a dual glucagon/glucagon-like peptide-1 (GCG/GLP-1) receptor agonist originally discovered by Eli Lilly and Co., of Indianapolis.

China clears mazdutide as first dual GCG/GLP-1RA for weight loss

Mon, 30 Jun 2025 11:00:00 -0400

Jyong’s $20M US IPO to help refile NDA of urological botanic drug

Tue, 24 Jun 2025 11:00:00 -0400

Jyong Biotech Ltd. raised $20 million from its Nasdaq debut June 17 to advance a pipeline of botanical drugs targeting male urinary disorders. The New Taipei City, Taiwan-headquartered company’s shares began trading under the ticker MENS, and closed at $10.11 apiece at the bell, up 34.80% from its listing price of $7.50 per share. Shares had kicked up to $15 at opening, reaching double its offering price.

Jyong’s $20M US IPO to help refile NDA of urological botanic drug

Wed, 18 Jun 2025 11:00:00 -0400

Oral GLP-1 Wegovy chases approval in obesity

Mon, 05 May 2025 11:00:00 -0400

The U.S. FDA has accepted the NDA for an oral formulation of Novo Nordisk A/S’s Wegovy (semaglutide) in treating obesity. If approved, the daily pill will be the first oral GLP-1 for treating chronic weight management. So far, however, it has been injectables leading the way to approval.

China’s NMPA accepts Innocare’s zurletrectinib NDA for solid tumors

Tue, 22 Apr 2025 12:00:00 -0400

China’s National Medical Products Administration (NMPA) has accepted Innocare Pharma Ltd.’s NDA for its second-generation pan-tropomyosin receptor kinase inhibitor, zurletrectinib (ICP-723), for treating adults and adolescents with advanced solid tumors harboring neurotrophic tyrosine receptor kinase gene fusions.

China’s NMPA accepts Innocare’s zurletrectinib NDA for solid tumors

Wed, 16 Apr 2025 12:00:00 -0400

Kelun posts phase I/II data of China’s first TROP2 ADC for NSCLC

Tue, 15 Apr 2025 11:00:00 -0400

Phase I/II trial results of sacituzumab tirumotecan (sac-TMT; Sichuan Kelun-Biotech Biopharmaceutical Co. Ltd.) published in Nature Medicine came a month after China’s National Medical Products Administration gave clearance to Kelun-Biotech’s supplemental NDA of sac-TMT to treat advanced non-small-cell lung cancer (NSCLC).