Pharma and biotech news, week of Sep 14 – Sep 20, 2026

The FDA approved a supplemental new drug application from IntraBio Inc for Aqneursa (levacetylleucine) oral suspension. The supplement provides for using the drug to treat ataxia in adults and pediatric patients weighing at least 15 kg with ataxia-telangiectasia. PDUFA goal date: 2026-09-19.AI summary

Ultragenyx received FDA approval for Fayuvi, a biologic (CBER). Application BLA125845. PDUFA goal date was 2026-09-19.

Viatris Inc. announced that Japan's Ministry of Health, Labour and Welfare approved WAKIX (pitolisant) for narcolepsy and excessive daytime sleepiness associated with obstructive sleep apnea syndrome. The decision makes WAKIX the first histamine H3 receptor antagonist/inverse agonist approved in Japan for these indications. The approval was backed by positive data from domestic Phase 3 trials in Japan and multiple international clinical studies.AI summary

Japan approved Eisai and Biogen's LEQEMBI Pen, a subcutaneous autoinjector formulation of lecanemab, for treating early Alzheimer’s disease. The approval provides a new once-weekly at-home administration option supporting efficacy comparable to biweekly intravenous dosing with lower injection-related reactions. At-home self-administration will be available following designated insurance listing and required training.AI summary

The FDA has approved Emerge Bioscience Pte. Ltd.'s biologics license application (BLA 761497/Original 2) for Garzulys (insulin aspart-fsan) injection. The action approves Garzulys as an interchangeable biosimilar product.AI summary

The FDA approved Telix's New Drug Application for Pixclara (floretyrosine F 18, or 18F-FET), a PET imaging drug to differentiate recurrent or progressive glioma from treatment-related change in adults and pediatric patients 1 month of age and older. The release calls it the first FDA-approved FET-PET imaging drug for glioma, noting that FET-PET was previously recommended in guidelines but not available as an FDA-approved product in the U.S. Approximately 24,000 new glioma cases are diagnosed in the U.S. each year.AI summary

The FDA has approved Biofrontera's supplemental New Drug Application for Ameluz topical gel, 10%, in combination with BF-RhodoLED light therapy for treating superficial Basal Cell Carcinoma in adults. The approval was based on Phase 3 trial data showing a 66% composite complete response rate with Ameluz PDT versus 5% with placebo-PDT. Biofrontera plans an official launch of the indication between late Q4 2026 and Q1 2027.AI summary

The CHMP has recommended EU approval for Alexion and AstraZeneca's Klygefa (gefurulimab) as an add-on therapy for adults with anti-AChR antibody-positive generalised myasthenia gravis. The positive opinion was based on the pivotal Phase 3 PREVAIL trial, which demonstrated a statistically significant improvement in MG-ADL scores at week 26 compared to placebo. Klygefa is also currently under regulatory review in the US, China, and other countries.AI summary

The CHMP has recommended AstraZeneca and Daiichi Sankyo’s Enhertu for EU approval as an adjuvant monotherapy for adult patients with HER2-positive early breast cancer with residual invasive disease following neoadjuvant treatment. The positive opinion is based on Phase 3 DESTINY-Breast05 trial data showing Enhertu reduced the risk of invasive disease recurrence or death by 53% versus T-DM1.AI summary

The US FDA accepted and granted Priority Review to Alexion and AstraZeneca's Biologics License Application for efzimfotase alfa to treat hypophosphatasia in patients aged 2 years and older. The application is supported by results from three Phase III trials: HICKORY, MULBERRY, and CHESTNUT. A regulatory decision is expected in the first half of 2027 under PDUFA.AI summary

Corcept Therapeutics announced that the CHMP recommended European Commission approval of relacorilant (Lifyorli), combined with nab-paclitaxel, for platinum-resistant ovarian cancer. The recommendation is supported by positive data from Phase 2 and pivotal Phase 3 ROSELLA trials. The European Commission is expected to issue its final decision in the fourth quarter of 2026.AI summary

Merck announced that the European Medicines Agency's CHMP adopted a positive opinion recommending KEYTRUDA in combination with Padcev as a perioperative treatment for adults with resectable muscle-invasive bladder cancer. The recommendation is supported by Phase 3 KEYNOTE-B15 trial data showing significant improvements in event-free survival, overall survival, and pathologic complete response rates. The European Commission will review the recommendation, with a final marketing authorization decision expected by Q4 2026.AI summary

The U.S. FDA has accepted Takeda's New Drug Application for zasocitinib under Priority Review to treat moderate-to-severe plaque psoriasis. The submission is backed by pivotal Phase 3 trials where about 70% of patients achieved clear or almost clear skin at week 16. A PDUFA target action date is scheduled for the first quarter of calendar year 2027.AI summary

Rocket Pharmaceuticals announced FDA alignment on continuing enrollment and dosing in the global pivotal Phase 2 trial of RP-A501 for Danon disease. The FDA confirmed a 12-patient pivotal population and 12-month co-primary endpoints intended to support potential accelerated approval. Rocket expects to complete dosing of the remaining nine patients by mid-2027.AI summary

Elevar Therapeutics has submitted a marketing authorization application to the European Medicines Agency for lirafugratinib as a second-line treatment for advanced/metastatic cholangiocarcinoma with FGFR2 fusions or rearrangements. The filing is based on the Phase 1/2 ReFocus study, which showed an objective response rate of 46.5%. Elevar will continue advancing regulatory processes in the U.S. and EU while expanding the drug's development in other solid tumors.AI summary

Revolution Medicines announced that the U.S. FDA granted Breakthrough Therapy Designation to RASONQUE (daraxonrasib) in combination with gemcitabine and nab-paclitaxel for treatment-naïve metastatic pancreatic adenocarcinoma. The designation was based on Phase 1/2 data from the RMC-GI-102 trial showing preliminary antitumor activity and manageable safety. These results informed the ongoing global Phase 3 RASolute 303 trial evaluating the combination.AI summary

Aquestive Therapeutics has resubmitted its New Drug Application to the FDA for Anaphylm (dibutepinephrine) sublingual film to treat Type I allergic reactions, including anaphylaxis. The resubmission follows a complete response letter and is supported by completed human factors and pharmacokinetic studies. The FDA will determine the resubmission classification and review timeline after its initial review.AI summary

The FDA has accepted Cogent Biosciences' New Drug Application for bezuclastinib to treat patients with Advanced Systemic Mastocytosis. The NDA is supported by data from the APEX trial demonstrating a 65% overall response rate. The FDA assigned a PDUFA target action date of June 29, 2027.AI summary

The U.S. FDA has approved an update to the Prescribing Information for Amgen's IMDELLTRA (tarlatamab-dlle). The label update reduces the recommended monitoring time for the first two doses to 6 to 8 hours, down from 22 to 24 hours. Amgen continues to evaluate reduced post-infusion monitoring timing in several ongoing studies across additional indications and lines of therapy.AI summary

Capricor Therapeutics announced it will present 24-month HOPE-3 and open-label extension data for Deramiocel in Duchenne muscular dystrophy at the 2026 World Muscle Society Congress. The presentations include late-breaking poster and oral presentations reporting skeletal muscle and cardiac outcomes. These data were submitted in a recent BLA amendment, which has a PDUFA action date of November 22, 2026.AI summary

Biogen announced the journal publication of adolescent subgroup results from the Phase 3 VALIANT study evaluating EMPAVELI in C3G or primary IC-MPGN. At Week 26, EMPAVELI-treated adolescents achieved a 75% relative reduction in proteinuria compared to placebo. The publication follows a recent FDA label update expanding EMPAVELI's indication in patients aged 12 and older.AI summary

Vera Therapeutics announced that TRUTAKNA (atacicept-vymj) met all prespecified endpoints in the Phase 3 ORIGIN 3 trial final efficacy analysis for primary IgA nephropathy. Treatment showed a statistically significant placebo-adjusted eGFR change of 5.6 mL/min/1.73m2 at 52 weeks and a 76% risk reduction in composite kidney disease progression through 104 weeks. The company plans to submit a supplemental BLA to the FDA in Q4 2026.AI summary

Summit Therapeutics presented updated overall survival results from the global Phase 3 HARMONi trial at WCLC 2026. In a June 2026 analysis, ivonescimab plus chemotherapy demonstrated an overall survival hazard ratio of 0.76 compared to placebo plus chemotherapy in the global intention-to-treat population, with consistent results in Western and Asian patients. Summit's BLA for ivonescimab in EGFR-mutated non-small cell lung cancer is under FDA review with a PDUFA action date of November 14, 2026.AI summary

Johnson & Johnson announced final overall survival results from the Phase 3 PAPILLON study of first-line RYBREVANT plus chemotherapy in advanced EGFR exon 20 insertion mutation-positive NSCLC. The combination delivered a median overall survival of 34.3 months compared to 27.9 months for chemotherapy alone. These data were presented at the 2026 World Conference on Lung Cancer.AI summary

Summit Therapeutics noted that partner Akeso presented updated overall survival data from the Phase 3 HARMONi-2 trial evaluating ivonescimab versus pembrolizumab in PD-L1-positive advanced NSCLC. Ivonescimab monotherapy demonstrated a statistically significant improvement in overall survival, achieving a median OS of 30.8 months compared to 22.6 months for pembrolizumab. Summit is currently recruiting patients for its global Phase 3 HARMONi-7 study.AI summary

Positive results from the DESTINY-Lung04 Phase III trial showed AstraZeneca and Daiichi Sankyo's Enhertu achieved a median progression-free survival of 14.3 months compared to 8.3 months for standard-of-care pembrolizumab plus chemotherapy in first-line HER2-mutant advanced non-small cell lung cancer. Enhertu reduced the risk of disease progression or death by 37.0% and demonstrated a 70.0% objective response rate. Overall survival testing will occur at future analyses.AI summary

BioNTech and OncoC4 presented updated data from stage 1 of the non-pivotal PRESERVE-003 Phase 3 trial of gotistobart at the IASLC 2026 World Conference on Lung Cancer. In 87 patients with metastatic squamous NSCLC, median overall survival was 18.5 months with gotistobart versus 10.0 months with docetaxel (HR: 0.56; nominal p-value=0.0295), at a data cut-off of July 17, 2026. The pivotal stage 2 portion is currently ongoing at more than 160 sites globally.AI summary

AstraZeneca announced updated exploratory results from the Phase III ADAURA trial evaluating Tagrisso in early-stage EGFR-mutated non-small cell lung cancer. At eight years of follow-up, Tagrisso reduced the risk of death compared to placebo by 47% in the primary population and 48% in the overall trial population. These data reinforce Tagrisso's role as the adjuvant standard of care.AI summary

Immunocore achieved its target enrollment of 540 patients in the registrational TEBE-AM trial evaluating KIMMTRAK (tebentafusp) for advanced melanoma. The trial evaluates tebentafusp as monotherapy and combined with pembrolizumab versus Investigator's Choice. Topline overall survival data from the trial are expected as early as the end of 2026.AI summary

Cullinan Therapeutics will host a virtual analyst and investor event today to discuss interim analysis results from the Phase 3 REZILIENT3 trial of zipalertinib plus chemotherapy. The data were presented at the IASLC 2026 World Conference on Lung Cancer. Company leadership will participate in the webcast.AI summary

Johnson & Johnson announced results from the Phase 2b COPERNICUS study evaluating subcutaneous RYBREVANT FASPRO plus LAZCLUZE in patients with previously untreated EGFR-mutated advanced NSCLC. Data presented at WCLC 2026 showed low rates of treatment-related events, with an 8% treatment discontinuation rate due to adverse events. The study continues to evaluate Cohort 1 towards a target enrollment of 300 patients.AI summary

Alveus Therapeutics has initiated the Phase 2 PRESERVE trial and dosed the first patient with ALV-100. The trial is evaluating ALV-100 for long-term weight management in approximately 220 adults who achieved at least 10% weight loss on semaglutide. Participants will be randomized across four dose regimens or placebo for 24 weeks.AI summary

Arrowhead Pharmaceuticals announced interim topline clinical data from its Phase 1/2a trial of ARO-DIMER-PA in mixed hyperlipidemia. Single doses achieved mean maximal reductions of 72% in PCSK9 and 88% in APOC3, as well as a 54% reduction in LDL-C. Additional details will be presented at an upcoming medical congress.AI summary

GSK presented positive registrational Phase 1/2 data for Jideytro (zidesamtinib) in TKI-naïve ROS1-positive non-small cell lung cancer patients. The drug demonstrated a 94% objective response rate with durable efficacy and low rates of treatment discontinuation. GSK plans to submit a supplemental New Drug Application to the US FDA in 2026 to support first-line expansion.AI summary

Jazz Pharmaceuticals has completed its acquisition of Actio Biosciences for $820 million upfront, making Actio a wholly-owned subsidiary. The transaction adds ABS-1230, a clinical-stage KCNT1 inhibitor for KCNT1-related epilepsy, to Jazz's rare disease pipeline. Jazz plans to advance the potential first-in-class treatment rapidly.AI summary

GSK has entered an agreement to acquire full global rights to a potential best-in-class trispecific T cell-engager programme for multiple myeloma from Chimagen Biosciences. The agreement has a total potential value of up to $750 million, including an upfront fee and success-based milestone payments. GSK plans to develop and commercialise the asset, with phase I trials expected to begin in 2027.AI summary

Biotechnology company. Total offering $70M; first sale 2026-08-26. Form D is filed up to 15 days after the first sale.

Sanofi and Cheplapharm intend to create a strategic partnership in mature medicines. Cheplapharm will take over 20 mature medicines, including Lovenox/Clexane, and three manufacturing sites worldwide from Sanofi. In return, Sanofi will receive a 26.4% equity stake in Cheplapharm, with the transaction expected to be fully completed by the third quarter of 2027.AI summary

Ascendis Pharma announced it will regain exclusive development, manufacturing, and commercialization rights to TransCon technology-based products in metabolic and cardiovascular diseases following the termination of its collaboration agreement with Novo Nordisk. Neither party has continuing financial obligations to the other. Ascendis plans to advance multiple programs, including once-monthly TransCon Semaglutide for obesity and type 2 diabetes.AI summary

Ascendis Pharma announced that its Board of Directors has authorized a share repurchase program. The company may repurchase up to $400 million of its ordinary shares. Repurchases will be determined based on market conditions, share price, and other factors.AI summary