Approvals, filings, CRLs, breakthrough designations and advisory committees.
JAMA reports that the FDA has approved a gene therapy for younger children with sickle cell disease. Only the headline was available; the full text was not captured. DOI: 10.1001/jama.2026.9488.
Why it matters: FDA approval of a gene therapy for younger sickle cell disease children
Ruidi Bio's SPA-C02 steep pulse ablation device has received FDA approval, completing regulatory clearance across China, Europe and the US. The tumour ablation system was the first of its kind to gain both NMPA's innovative device green channel and FDA breakthrough device designation, received NMPA market approval in China in late 2024, and then entered Europe. The company plans to build on this triple certification for global expansion.
Why it matters: FDA approval completes the device's regulatory coverage of the three main medical markets.
The FDA has approved BMS's Iberdomide (brand name Zenbexus), the first approved cereblon E3 ubiquitin ligase modulator (CELMoD) therapy. It is approved with subcutaneous daratumumab and hyaluronidase-fihj plus dexamethasone (ZDd) for adults with multiple myeloma who have received at least one prior line of therapy. Approval rests on the Phase 3 EXCALIBER-RRMM trial, which compared ZDd against daratumumab, bortezomib and dexamethasone (DVd) in relapsed or refractory multiple myeloma.
Why it matters: First FDA approval of a cereblon E3 ligase modulator, a new protein degrader class, for multiple myeloma.
The FDA granted accelerated approval on August 13, 2026 to iberdomide in combination with daratumumab-hyaluronidase fihj and dexamethasone (IberDd) for adults with multiple myeloma who have received at least one prior line of therapy including a proteasome inhibitor and an immunomodulatory agent. Iberdomide is the first approved CELMoD drug for multiple myeloma, designed around the ubiquitin-proteasome system recognised by the 2004 Nobel Prize in Chemistry.
Why it matters: First CELMoD class drug approved for multiple myeloma, an approval relevant to clinicians treating relapsed patients.
The FDA has approved vepdegestrant (Veppanu), an estrogen receptor degrader developed by Arvinas and Pfizer, for a specific subgroup of patients with advanced or metastatic breast cancer. It is the first approved PROTAC, a modality that degrades rather than inhibits its target, marking a milestone for targeted protein degradation.
Why it matters: First FDA approval of a PROTAC validates targeted protein degradation as a drug modality.
The FDA has approved Bristol-Myers Squibb's iberdomide, a new CELMoD drug, for relapsed or refractory multiple myeloma. Approval is based on the EXCALIBER-RRMM trial, in which the iberdomide regimen doubled the rate of minimal residual disease-negative complete response versus daratumumab plus bortezomib and dexamethasone, 41% versus 21%. The company says it will continue exploring broader treatment options for multiple myeloma patients.
Why it matters: FDA approval of a first-in-class CELMoD drug for relapsed or refractory multiple myeloma, backed by MRD-negative complete response data.
The FDA has granted accelerated approval to Bristol Myers Squibb's iberdomide (brand name Zenbexus), the first CELMoD-class drug to receive FDA approval. It is approved in combination with daratumumab and dexamethasone for adults with relapsed or refractory multiple myeloma who have received at least one prior line of therapy. Iberdomide works through targeted protein degradation, a mechanism distinct from traditional IMiDs such as lenalidomide and pomalidomide. Bristol Myers Squibb announced the approval on August 13, 2026.
Why it matters: First FDA approval of a CELMoD-class drug gives relapsed or refractory myeloma patients a new option.
The FDA has approved BMS's iberdomide for multiple myeloma, making it the first approved CELMoD drug, a new class of cereblon-directed protein degraders. The launch addresses declining sales of BMS's older immunomodulators, with Pomalyst revenue down 48% and Revlimid down 56% in the first half of this year.
Why it matters: FDA approval of the first-in-class CELMoD drug replaces two declining BMS multiple myeloma franchises.
The MHRA approved vimseltinib (Romvimza) on 14 August 2026 to treat adults with tenosynovial giant cell tumour where the disease impacts movement or surgery is not an option. The marketing authorisation went to Deciphera Pharmaceuticals via the International Recognition Procedure Route B. The capsule is taken twice a week and slows tumour growth by blocking proteins that drive it; the product is subject to additional monitoring and contraindicated in pregnancy.
Why it matters: UK regulator adds a non-surgical systemic option for a rare joint tumour with few alternatives.
The FDA approved Bristol Myers Squibb's oral drug iberdomide, branded Zenbexus, for advanced multiple myeloma in patients who have received at least one prior line of therapy. It is the first drug in a new class of medicines for the blood cancer and the first cleared using a more sensitive measure of remission. Zenbexus will be used in combination with Darzalex and dexamethasone, and Bristol sees the class opening possibilities for more combinations.
Why it matters: First approval of a novel drug class for multiple myeloma, with a new remission standard used by FDA.
The European Commission has revoked the EU marketing authorization for Tavneos, used for active granulomatosis with polyangiitis and microscopic polyangiitis in ANCA-associated vasculitis, adopting a June CHMP recommendation. Regulatory documents detail 'serious breaches' of good clinical practice in the pivotal phase 3 Advocate study, where sponsor personnel allegedly saw unblinded efficacy data after database lock and re-adjudicated nine patients, turning a non-significant result into statistical significance at week 52. CHMP also cited fatal cases of drug-induced liver injury and vanishing bile duct syndrome. CSL Vifor, the EU marketing operator, will implement the withdrawal, while Amgen, which bought developer ChemoCentryx for $3.7 billion in 2022, disagrees and continues fighting a separate FDA withdrawal request in the U.S.
Why it matters: EU regulators detail data-integrity breaches behind the Tavneos withdrawal while Amgen contests parallel U.S. action.
The FDA has granted accelerated approval to Bristol Myers Squibb's Zenbexus (iberdomide), the first cleared CELMoD therapy, in combination with Johnson & Johnson's Darzalex and dexamethasone for adults with previously treated multiple myeloma. The approval rests on the phase 3 Excaliber-RRMM trial, where the regimen achieved an MRD-negative complete response rate of 41% versus 21% for Darzalex, Velcade and dexamethasone at a median follow-up of 16 months. BMS must provide confirmatory PFS data expected this year, and its second CELMoD, mezigdomide, faces an FDA decision date of May 13, 2027.
Why it matters: First approval for a CELMoD drug and first new myeloma drug cleared directly on MRD as an endpoint.
The FDA has approved Bristol Myers Squibb's Iberdomide (Zenbexus) in combination with daratumumab and dexamethasone for patients with relapsed or refractory multiple myeloma. The approval, announced on August 13, makes Iberdomide the first cereblon E3 ubiquitin ligase modulator (CELMoD) therapy cleared by the FDA and the first product from BMS's CELMoD pipeline.
Why it matters: First FDA approval of a CELMoD therapy and of BMS's CELMoD pipeline in relapsed or refractory multiple myeloma.
On 12 August 2026, China's NMPA approved Akeso's supplemental new drug application for ivonescimab injection combined with chemotherapy as first-line treatment of advanced squamous non-small cell lung cancer. It is ivonescimab's third approved lung cancer indication. The regimen, supported by the Phase 3 HARMONi-6/AK112-306 study, is described as the first to show significant benefits in both progression-free survival and overall survival versus an anti-PD-1 antibody plus chemotherapy in a randomized, double-blind trial.
Why it matters: NMPA approval of a first-line squamous NSCLC indication backed by dual PFS and OS superiority over PD-1 plus chemotherapy.
The US FDA has granted accelerated approval to Bristol Myers Squibb's Zenbexus (iberdomide) in combination with subcutaneous daratumumab and dexamethasone (ZDd) for adults with multiple myeloma who have received at least one prior line of therapy. Zenbexus is the first cereblon E3 ubiquitin ligase modulator (CELMoD) therapy approved by the FDA. The approval is based on results from the phase 3 EXCALIBER-RRMM trial.
Why it matters: First FDA approval of a CELMoD protein-degrader class therapy in relapsed multiple myeloma.
I-Mab and Biogen have secured China's approval of felzartamab, marking the product's first approval anywhere in the world. Felzartamab is a humanized monoclonal antibody targeting CD38, a protein mainly expressed on plasma cells, plasmablasts and natural killer cells, and selectively clears CD38-positive plasma cells. The companies position it as a potential first-in-class therapy across multiple immune-mediated diseases.
Why it matters: First global approval for a CD38-targeting antibody developed by I-Mab and Biogen.
China's National Medical Products Administration has approved fizzeftamab for injection (brand name Jingfei), filed by I-Mab Biopharma (Hangzhou), for adult multiple myeloma patients who have received at least one prior line of therapy. The drug is to be used in combination with lenalidomide and dexamethasone, giving these patients a new treatment option.
Why it matters: NMPA approval of a new second-line-plus multiple myeloma therapy from I-Mab.
The UK's MHRA completed its review and granted marketing authorisation on August 10 for Lilly's oral, non-peptide small-molecule GLP-1 drug Orforglipron, brand name Foundayo. It becomes the first oral agent of its type approved in Europe with dual indications for weight reduction and glucose lowering, with few restrictions on how the tablet is taken.
Why it matters: First oral dual-indication GLP-1 approval in Europe reshapes the regional obesity market.
China's NMPA has approved Akeso's ivonescimab injection plus chemotherapy as first-line treatment for advanced squamous non-small cell lung cancer, the drug's third approved lung cancer indication. The approval rests on the Phase 3 HARMONi-6/AK112-306 study of 532 patients, which met all primary and secondary endpoints with statistically significant PFS and OS benefit versus PD-1 antibody plus chemotherapy and a good safety profile; OS results were presented at the 2026 ASCO plenary session and published in The Lancet.
Why it matters: NMPA approval of a first-in-class cancer immunotherapy in first-line squamous NSCLC sets a new treatment standard.
China's NMPA has approved registration applications for several innovative medical devices, including a toric trifocal intraocular lens from Henan Saimeishi Biotechnology, a cardiac pulsed-field ablation system and single-use catheter from Shenzhen Benex Medical, and a transcatheter tricuspid clip system from Hangzhou Duanyou Medical; some are first-in-China products. The trifocal toric lens is a foldable one-piece posterior-chamber lens for correcting aphakia in adults with cataracts and corneal astigmatism, aiming to improve near, intermediate and distance vision while reducing dependence on glasses.
Why it matters: NMPA approvals of multiple innovative devices, including first-in-China products, affect device makers and clinicians.