Simcere's Kocela (trilaciclib hydrochloride for injection) won NMPA approval on August 24 for a new indication: reducing chemotherapy-induced myelosuppression in relapsed extensive-stage small cell lung cancer patients before topotecan-containing regimens. The CDK4/6 inhibitor, in-licensed from G1 Therapeutics, now covers bone marrow protection across first-line and second/third-line chemotherapy for extensive-stage SCLC, making it the only drug approved in China for the full treatment course.
On August 19, 2026, the FDA granted accelerated approval to Genglycos (pariglasgene brecaparvovec-opnr) for patients aged 8 and older with glycogen storage disease type Ia (GSDIa). It is the first approved therapy for this rare inherited disease and a one-time AAV8 gene therapy approved on a surrogate endpoint. The approval comes as the FDA tightens its review standards for cell and gene therapies.
The US FDA has approved Regeneron's BLA for garetosmab, a monoclonal antibody, to treat adults with fibrodysplasia ossificans progressiva (FOP), a progressive ultra-rare genetic disease characterised by heterotopic ossification. The approval was announced on August 19. Per the Insight database, garetosmab is the first approved activin A-targeted therapy worldwide.
China's National Medical Products Administration has approved two innovative drugs: subcutaneous anfuliqita monoclonal injection, brand name Yisaina, applied for by a Shanghai-based pharmaceutical company, for acute attacks of gouty arthritis in adults for whom NSAIDs and/or colchicine are contraindicated, not tolerated or ineffective, and who are unsuitable for repeated steroid use; and Hengyouda (likangopan fumarate capsules), a class 1 innovative drug from Chengdu Shengdi Pharmaceutical, granted through priority review for adults with paroxysmal nocturnal hemoglobinuria (PNH) who have not previously received complement inhibitor therapy.
China's NMPA has approved Hengrui Pharmaceuticals' Class 1 innovative drug fumarate likanuopan capsules via priority review for adult patients with paroxysmal nocturnal hemoglobinuria (PNH) who have not previously received complement inhibitor treatment. The oral complement factor B (CFB) inhibitor offers an alternative to intravenous C5 antibody eculizumab, which controls intravascular hemolysis but leaves many patients with extravascular hemolysis, anemia, and a heavy treatment burden from repeated infusions.
NMPA approved Hengrui Medicine's licankelpar fumarate capsules (development code HRS-5965), a self-developed class 1 oral small-molecule inhibitor of complement factor B, via priority review on August 20 for adults with paroxysmal nocturnal hemoglobinuria (PNH) who have not previously received complement inhibitor treatment. The trial enrolled 78 such patients randomized 1:1 to oral licankelpar 50 mg twice daily or intravenous eculizumab for 24 weeks.
China's NMPA approved on August 20 the marketing of Sunsum Pharmaceutical's SinoSun Guojian-developed SSGJ-613 (brand name Yisaina), a subcutaneous recombinant humanized anti-IL-1β monoclonal antibody, under the generic name anfrilitamab injection. The drug treats acute gouty arthritis attacks in adults for whom NSAIDs and colchicine are contraindicated, poorly tolerated or ineffective, and who are unsuitable for repeated corticosteroid use. It works by specifically blocking IL-1β, a key inflammatory mediator in acute gout flares.
China's National Medical Products Administration has approved likangkepan fumarate capsules, brand name Hengyouda, a Class 1 innovative drug filed by Chengdu Shengdi Pharmaceutical, via priority review. The indication is adults with paroxysmal nocturnal hemoglobinuria (PNH) who have not previously received complement inhibitor treatment. The approval adds a new treatment option for these patients.
FDA has approved OncoSil Medical's phosphorus-32 (P-32) radioactive implant device through the Humanitarian Device Exemption pathway for unresectable, non-metastatic distal bile duct cancer. It is described as the first and so far only Class III medical device approved specifically for this rare, aggressive bile duct cancer subtype. The approval adds a new radiotherapy option delivered via an established isotope rather than newer agents such as lutetium-177 or actinium-225.
China's National Medical Products Administration has approved Sunshine Guojian Pharmaceutical's Shanghai-applied anfuliqita monoclonal antibody injection for subcutaneous use, brand name Yisaina, for acute gouty arthritis attacks in adults. The indication covers patients for whom NSAIDs and/or colchicine are contraindicated, not tolerated or ineffective, and those unsuited to repeated steroid use. The approval adds a new treatment option for these patients.
China's NMPA has approved Anfuliqita (brand name Yisaina), a subcutaneous injection from Sunshine Guojian Pharmaceutical (Shanghai), for acute gouty arthritis attacks in adults for whom NSAIDs and/or colchicine are contraindicated, not tolerated or ineffective, and who are unsuitable for repeated steroid use. It is Shanghai's fifth domestically developed Class 1 innovative drug approved this year and Pudong's fourth. The drug, developed in-house, is also indicated for moderate-to-severe plaque psoriasis in adults suited to systemic therapy or phototherapy.
China's NMPA has approved SSGJ-613 (anfuliqitamab injection, subcutaneous), a recombinant humanized anti-IL-1β monoclonal antibody developed by Sunshine Guojian, a subsidiary of Sunshine Pharma. The approval covers acute flares of gouty arthritis in adults for whom NSAIDs and/or colchicine are contraindicated, not tolerated or ineffective, and who are unsuitable for repeated corticosteroid use. SSGJ-613 binds IL-1β, a key mediator of acute gout flares, blocking its signalling and inflammatory effects.
The FDA granted accelerated approval to Ultragenyx's AAV gene therapy pariglasgene brecaparvovec-opnr, brand name Genglycos, for adults and children over 8 with glycogen storage disease type Ia. In the phase 3 GlucoGene study, the one-time AAV8 therapy cut daily cornstarch intake by a mean 31% from baseline versus placebo over 48 weeks. Ultragenyx, which also received a priority review voucher, must provide two years of safety and efficacy data on 50 treated and 20 control patients; it awaits a Sept. 19 decision on UX1111.
The FDA has approved the AAV gene therapy Genglycos (pariglasgene brecaparvovec-opnr, development code DTX401) for glycogen storage disease type Ia, a rare inherited metabolic disorder caused by G6PC gene mutations leading to glucose-6-phosphatase deficiency. It is the first gene therapy the FDA has approved for this disease.
The FDA granted accelerated approval to Ultragenyx Pharmaceutical's Genglycos (pariglasgene brecaparvovec-opnr), a one-time AAV8 gene therapy and the first approved treatment for glycogen storage disease type Ia in patients aged 8 and older. In a 48-week randomized placebo-controlled study, patients achieved a statistically significant 31% mean reduction in daily cornstarch intake, the primary endpoint. The manufacturer must complete additional trials to confirm effectiveness, and the label warns of anaphylaxis, liver toxicity, adrenal insufficiency and tumorigenicity risk.
The FDA has approved Regeneron Pharmaceuticals' Pasatru for fibrodysplasia ossificans progressiva, an ultra-rare disease in which bone grows where it should not. The approval caps a three-decade effort and follows a pivotal trial in which the medicine drastically cut the risk of new bone formation. Patients with FOP typically rely on wheelchairs by age 25, and investigators hope Pasatru can help maintain mobility.
On August 6, 2026, the FDA granted accelerated approval to Replimune's oncolytic virus therapy Tudriqev in combination with a PD-1 inhibitor for patients with PD-1-resistant advanced melanoma. It is the first approved oncolytic virus combination therapy worldwide and the first oncolytic virus approval by the FDA in 11 years.
China's regulator has approved AstraZeneca's human monoclonal antibody targeting TSLP for two indications at once: maintenance treatment of severe asthma in adults and adolescents over 12, and, with nasal corticosteroids, chronic rhinosinusitis with nasal polyps in adults inadequately controlled by systemic corticosteroids or surgery. It is the first severe asthma biologic in China unrestricted by phenotype or biomarker level and the first TSLP-targeting drug for CRSwNP.
FDA has approved Lantheus's tau PET tracer TAUKLARIFY. The approval rests on two blinded-read studies covering PET images from more than 500 subjects across three trials of cognitively normal, mildly cognitively impaired and mild Alzheimer's disease participants. Safety was assessed in 1734 subjects, with headache (0.7%) the most common adverse reaction at rates of 0.1% or more. In 2023, Lantheus's partner Xontong Pharmaceutical out-licensed all ex-China rights to the tracer, then code-named XTR006/MK-6240, through a US joint venture while retaining exclusive rights to develop, manufacture and commercialize it in mainland China, where it is now in Phase III trials.
The FDA has approved Regeneron's Pasatru (garetosmab), an intravenous anti-activin A monoclonal antibody given every four weeks, for fibrodysplasia ossificans progressiva in adults. The approval rests on the phase 3 Optima trial, in which 63 adults were randomized and the 10- and 3-mg/kg doses cut new bone lesions by 90% and 94% versus placebo over 56 weeks, with high dose reducing flare-ups by 89%. Pasatru becomes the second FOP drug after Ipsen's Sohonos and will reach roughly 220 U.S. adult patients; Regeneron declined to state a list price but plans co-pay support and free medicine for eligible patients.