Pfizer Canada and BioNTech announced on Thursday, September 17, 2026 that Health Canada authorized the XFG variant-adapted Comirnaty vaccine for active immunization against COVID-19 in people aged 6 months and older. The authorization was based on the cumulative clinical, non-clinical, and real-world data the companies had already submitted. The companies said the vaccine will be available through various distribution channels in the coming days and weeks, and Pfizer Canada said it has enough inventory to meet its contractual commitments.
Roche announced on Thursday, September 17, 2026 that the Phase 3 CELESTIMO trial met its primary endpoint: Lunsumio (mosunetuzumab) plus lenalidomide produced a statistically significant improvement in progression-free survival versus rituximab plus lenalidomide in people with relapsed or refractory follicular lymphoma who had received at least one prior line of treatment. Overall survival data were immature at the interim analysis, and safety was consistent with the known profiles of both drugs, with no new signals. CELESTIMO is the confirmatory study required to convert the accelerated approval and conditional authorization of Lunsumio monotherapy for third-line or later follicular lymphoma into full approval. Roche will submit the data to health authorities and present them at an upcoming medical meeting.
Nuvation Bio announced on Thursday, September 17, 2026 that the FDA approved a supplemental New Drug Application updating the IBTROZI (taletrectinib) label for locally advanced or metastatic ROS1-positive non-small cell lung cancer. The label now reports a median duration of response of 49.7 months in TKI-naive patients in the Phase 2 TRUST-I study, based on a median follow-up of 51 months and an August 2025 data cutoff that added 14 months of data from TRUST-I and TRUST-II. Nuvation said the approval came four months ahead of the PDUFA date and brought no changes to the label's safety sections. IBTROZI, which Nuvation describes as a CNS-active next-generation ROS1 inhibitor, was first approved on June 11, 2025; Innovent Biologics markets it in China as DOVBLERON.
Bristol Myers Squibb has told Orum Therapeutics it is ending development of BMS-986497 (ORM-6151), a degrader-antibody conjugate that pairs a CD33-targeting antibody with a GSPT1 degrader, after reviewing Phase 1 data in acute myeloid leukemia and high-risk myelodysplastic syndrome, Korea Biomedical Review reported on September 18, 2026. BMS bought the program from Orum in October 2023 for $100 million upfront in a deal worth up to $180 million; Orum keeps the upfront payment but is no longer eligible for up to $80 million in remaining milestones. Orum said it was notified unexpectedly and is assessing the situation. It will concentrate on ORM-1153, a CD123-targeting degrader-antibody conjugate for the same indications, for which the FDA has cleared an investigational new drug application.
Ultragenyx Pharmaceutical announced on Thursday, September 17, 2026 that the FDA granted standard full approval to FAYUVI (rebisufligene etisparvovec-hopf), a single-dose intravenous AAV9 gene therapy, for the neurologic manifestations of mucopolysaccharidosis type IIIA (MPS IIIA, Sanfilippo syndrome type A) in pediatric patients with preserved neurodevelopmental function. The company calls it the first FDA-approved treatment for Sanfilippo syndrome type A and its second gene therapy approval. Treated children (n=17) had a 23.5-point higher cognitive score than natural-history controls (n=27), with follow-up of up to nearly 8 years and reduced cerebrospinal fluid heparan sulfate. Ultragenyx received a priority review voucher, expects to ship within 30 to 60 days, and makes FAYUVI in Bedford, Massachusetts and at Andelyn Biosciences in Columbus, Ohio. BioSpace reported a $3.95 million wholesale acquisition cost, an initial FDA rejection in July 2025 over manufacturing issues, and a 13% share gain on the day.
Dualitas Therapeutics announced on Thursday, September 17, 2026 a research collaboration and license agreement with Roche to discover bispecific antibodies for immunology and inflammation diseases. Dualitas receives $36.5 million upfront and can earn research, development, and commercial milestones plus tiered royalties, for a total potential value of up to $1 billion. It will use its DualScreen Bispecific Discovery Engine to functionally screen more than 300,000 novel bispecific combinations, and Roche will handle all later preclinical development, regulatory, manufacturing, and commercial work.
NICE published final draft guidance on Thursday, September 17, 2026 recommending Enhertu (trastuzumab deruxtecan, from Daiichi Sankyo and AstraZeneca) for adults with HER2-low advanced or metastatic breast cancer whose disease has progressed after treatment, a group NICE estimated at about 1,000 people. NICE had previously rejected the drug for this use on cost-effectiveness grounds. It said the reversal followed an agreed commercial solution, the higher cost-effectiveness thresholds it began using in April 2026, and a new method for assessing quality of life published on August 27, 2026. The guidance is open to appeal until 5pm on October 7, 2026.
Vera Therapeutics reported on Tuesday, September 15, 2026 final efficacy results from the Phase 3 ORIGIN 3 trial of TRUTAKNA (atacicept-vymj) in 428 adults with primary IgA nephropathy at risk of progression. At 52 weeks, mean eGFR changed by -0.1 mL/min/1.73m² on TRUTAKNA versus -5.7 on placebo, and the annualized eGFR slope through 104 weeks was -0.6 versus -5.6 mL/min/1.73m² per year. TRUTAKNA cut the risk of composite kidney disease progression by 76% (hazard ratio 0.24; 11 versus 38 events), with no dialysis, transplant, or death events versus 8 on placebo, and proteinuria, galactose-deficient IgA1, and hematuria also fell significantly. Safety was generally comparable to placebo. Vera plans to submit a supplemental BLA for full approval in the fourth quarter of 2026 and reported more than 350 patient start forms in the first ten weeks of launch.
Cogent Biosciences (NASDAQ: COGT) announced Tuesday September 15, 2026 that the FDA accepted the New Drug Application (NDA) for bezuclastinib (a selective oral tyrosine kinase inhibitor targeting KIT D816V) in patients with Advanced Systemic Mastocytosis (AdvSM), an aggressive form of the rare hematologic disorder driven by activating mutations in the KIT receptor. FDA assigned a PDUFA target action date of June 29, 2027. The agency communicated that no advisory committee is planned and no potential review issues have been identified. Application support: the APEX Phase 2 registration trial primary endpoint reported a 65% overall response rate per modified International Working Group Myeloproliferative Neoplasms Research and Treatment / European Competence Network on Mastocytosis (mIWG-MRT-ECNM) criteria as of the March 31, 2026 data cutoff, including 57% of patients achieving complete response (CR), CR with partial hematologic recovery (CRh), or partial response (PR) as best response. Cogent had earlier received FDA acceptance for the bezuclastinib NonAdvSM (non-advanced systemic mastocytosis) NDA in March 2026 supported by the SUMMIT Phase 3 trial. Bezuclastinib would compete against Blueprint Medicines' Ayvakit (avapritinib) in the AdvSM segment; both target KIT D816V but Cogent has emphasized bezuclastinib's cleaner off-target profile and better CNS penetration profile.
Corbus Pharmaceuticals (NASDAQ: CRBP) announced Monday September 14, 2026 positive topline results from the Phase 1b CANYON-1 trial evaluating CRB-913 (once-daily oral peripherally-restricted CB1 inverse agonist for obesity) in 254 obese non-diabetic U.S. adults across 15 sites (NCT07310901). Mean weight loss at 12 weeks: 5.0% at 60 mg (n=62), 3.3% at 40 mg (n=61), 2.8% at 20 mg (n=65) versus 0.0% on placebo (n=66) — all p<0.0001. No plateau was observed at any dose. Discontinuation rates ranged 3.1-13.1% across doses (comparable to 6.9-20.7% for oral GLP-1s in cross-trial comparison). At the 60 mg dose: nausea 22.6%, diarrhea 22.6%, constipation 4.8%, vomiting 1.6%. Psychiatric adverse events at 60 mg: depression 1.6%, anxiety 4.8%, irritability 9.7%, insomnia 0% — no serious psychiatric events or suicidality reported. CRB-913 was designed with approximately 15-fold lower brain penetration than monlunabant in preclinical models to preserve CB1-driven weight loss while limiting the psychiatric side effects that led to the 2008 withdrawal of Sanofi's Acomplia (rimonabant). CRBP shares closed up 12% on the day. Next steps: FDA clinical development plan engagement, Phase 2 monotherapy trial initiation in H1 2027, evaluation of a GLP-1 combination approach, and full data presentation at ObesityWeek 2026 (November 14-17, Washington DC).
Insmed Incorporated (NASDAQ: INSM) presented Monday September 14, 2026 at 2:35 p.m. ET at the Morgan Stanley 24th Annual Global Healthcare Conference in New York, raising full-year 2026 BRINSUPRI (brensocatib, oral dipeptidyl peptidase 1 inhibitor approved August 2025 for non-cystic fibrosis bronchiectasis in ages 12+) revenue guidance to $1.25-1.4 billion, up from earlier launch-year projections. Q2 2026 BRINSUPRI revenue was $309.2 million (49% sequential growth over Q1) driven by strong U.S. launch momentum through specialty pharmacy channels. Management flagged peak U.S. sales potential above $7 billion in the bronchiectasis indication alone, before considering additional expansion into COPD or comorbid asthma populations. The Phase 3 ASPEN trial supporting the U.S. approval enrolled 1,721 NCFB patients across 35+ countries; brensocatib reduced pulmonary exacerbations by 21.2% at the 10 mg dose (primary endpoint met). EMA and MHRA reviews are ongoing with Japanese filing planned for H2 2026. Insmed also confirmed the September 6-9 ERS Congress presentations included the ASPEN post-hoc bronchiectasis-plus-prior-NTM analysis and a COMPERA 2.0 risk-assessment analysis of treprostinil palmitil inhalation powder (TPIP) in pulmonary arterial hypertension.
Alnylam Pharmaceuticals (NASDAQ: ALNY) presented Monday September 14, 2026 at 10:00 a.m. ET at the Morgan Stanley 24th Annual Global Healthcare Conference in New York with CEO Yvonne Greenstreet and John Kennedy (SVP Global Commercialization / TTR Franchise Lead), disclosing a modest trim to full-year TTR franchise revenue guidance as early AMVUTTRA (vutrisiran, subcutaneous RNAi TTR silencer approved March 2025 for adults with ATTR amyloidosis with cardiomyopathy) launch demand normalized. Management emphasized the underlying market remains large and underdiagnosed, physician adoption is strong, and pipeline funding from AMVUTTRA commercial success is not at risk. AMVUTTRA competes in ATTR-CM against Pfizer's Vyndaqel/Vyndamax (tafamidis, oral small-molecule stabilizer) plus the recently launched BridgeBio Attruby (acoramidis, oral) — the launch mix has shifted as switch-from-tafamidis patients and treatment-naive patients balance out. HELIOS-B Phase 3 data anchored the approval with a 33% reduction in the composite of all-cause mortality plus recurrent cardiovascular events at 36 months. The trim adds nuance to the Alnylam commercial story that had run largely positive through Q2 2026 earnings; investors focused on the deeper pipeline (nucresiran next-generation TTR silencer plus the broader RNAi platform) rather than the near-term guidance move.
Sichuan Kelun-Biotech Biopharmaceutical (HKEX: 6990) announced Monday September 14, 2026 that Phase 3 registrational study results for trastuzumab botidotin — a novel HER2-targeted antibody-drug conjugate — versus T-DM1 (Roche's Kadcyla, trastuzumab emtansine) in HER2-positive unresectable or metastatic breast cancer have been published in the Journal of Clinical Oncology. Trastuzumab botidotin produced 11.1 months median progression-free survival versus 4.4 months on T-DM1, a hazard ratio of 0.39 (61% reduction in risk of progression or death). Co-senior authors: Professors Xichun Hu and Hongxia Wang of Fudan University Shanghai Cancer Center, and Dr. Junyou Ge. The NMPA approved trastuzumab botidotin in October 2025 for adults with unresectable or metastatic HER2-positive breast cancer who had received at least one prior anti-HER2 therapy. The T-DM1 comparator (approved 2013) has historically been the standard of care in this second-line setting, though Daiichi Sankyo/AstraZeneca's Enhertu (trastuzumab deruxtecan) has since taken market share on stronger DESTINY-Breast trial data. Kelun-Biotech has opened a Phase 2 study of trastuzumab botidotin in patients who previously received a topoisomerase inhibitor payload ADC (which includes Enhertu-treated patients). The Phase 3 result strengthens the case for global development beyond China.
The Ultragenyx (NASDAQ: RARE) UX111 (rebisufligene etisparvovec, AAV9-delivered gene therapy for the SGSH gene) FDA PDUFA action date for Sanfilippo Syndrome Type A (mucopolysaccharidosis type IIIA, or MPS IIIA — a rare autosomal recessive lysosomal storage disorder causing progressive irreversible neurodegeneration in young children with a median life expectancy in the mid-teens) is set for Saturday September 19, 2026 (weekend PDUFA date; the FDA typically issues weekend action-date decisions on the adjacent Friday or the following Monday), five days from the September 14 opening of Morgan Stanley conference week. The resubmitted Biologics License Application under Accelerated Approval was accepted by the FDA in April 2026 following a July 2025 complete response letter that identified chemistry, manufacturing, and controls (CMC) plus manufacturing-site inspection issues. The clinical package includes up to 8 years of follow-up in treated children, with sustained cerebrospinal fluid heparan sulfate (the accumulated substrate) reduction plus preserved developmental trajectories relative to untreated natural history. If approved, UX111 would become the first-ever therapy for MPS IIIA anywhere in the world; the drug would also confer a Rare Pediatric Disease Priority Review Voucher on Ultragenyx (historically valued $150-350 million on the secondary market). Manufacturing runs domestically at Andelyn Biosciences in Columbus, Ohio and Ultragenyx's own Bedford, Massachusetts facility.
Corbus Pharmaceuticals (NASDAQ: CRBP) enters Sunday September 13, 2026 the final day of the countdown to Monday September 14 at 8:00 a.m. EDT conference call disclosing Phase 1b CANYON-1 topline data for CRB-913 (once-daily oral peripherally-restricted CB1 inverse agonist for obesity). The 16-week double-blind placebo-controlled dose-ranging study enrolled 240 obese non-diabetic U.S. adults across once-daily doses of 20 mg, 40 mg, and 60 mg (titrated from 20 mg over four weeks) with a 4-week safety follow-up (NCT07310901). Harold Bays MD (investigator) joins Corbus management. Investor focus is on the CB1 inverse agonist class safety-versus-efficacy trade-off: peripheral restriction (approximately 15-fold lower brain penetration than monlunabant in preclinical models) is designed to preserve dose-responsive weight loss while limiting the psychiatric side effects that led to the 2008 withdrawal of Sanofi's Acomplia (rimonabant). Corbus previously reported Phase 1a mean 2.9% placebo-adjusted weight loss by Day 14. Clean tolerability plus dose-responsive weight loss would enable Phase 2 initiation and position CRB-913 as one of the earliest non-incretin oral obesity options alongside Novo Nordisk cagrilintide (amylin), Structure Therapeutics aleniglipron (oral small-molecule GLP-1), and the Roche petrelintide-enicepatide combo Phase 2.
Pharming Group N.V. (NASDAQ: PHAR) announced Friday September 11, 2026 that the FDA approved the company's supplemental new drug application (sNDA) for Joenja (leniolisib, an oral selective phosphoinositide 3-kinase delta (PI3Kδ) inhibitor) at 40 mg and 50 mg twice-daily dosing for children aged 4 to 11 years weighing at least 27 kg with activated phosphoinositide 3-kinase delta syndrome (APDS). Joenja becomes the first FDA-approved treatment for children aged 4-11 with APDS, a rare primary immunodeficiency driven by gain-of-function mutations in the PIK3CD or PIK3R1 genes producing constitutive PI3Kδ pathway activation and recurrent sinopulmonary infections. Approval follows the February 2026 complete response letter, which required additional supportive data. The newly approved doses will be available through Pharming's specialty distribution network and patient-support infrastructure starting October 2026. Joenja was originally approved in March 2023 for APDS patients aged 12 and older; the pediatric label expansion extends the treated population substantially.
Mabwell Biosciences presented at the European Respiratory Society Congress 2026 in Barcelona (September 5-9, 2026) — with syndicated press coverage continuing through Saturday September 12 — Phase 1b/2a results for 9MW1911, an anti-ST2 monoclonal antibody developed on Mabwell's B-lymphocyte screening platform in China. In the 9MW1911-C03 study of former smokers with moderate-to-severe COPD, the annualized rate of moderate-to-severe exacerbations was reduced by 81% at the 900 mg dose and 24% at the 600 mg dose versus placebo; annualized severe exacerbation rate reductions were 100% and 47% respectively. The Phase 3 trial is expected to initiate around end of 2026 in China; the FDA has cleared the IND application for a Phase 2a U.S. trial. 9MW1911 targets ST2 (the IL-33 receptor) — the same biological pathway targeted by Regeneron/Sanofi's itepekimab and GSK's depemokimab in COPD. Sanofi and Regeneron's Phase 3 AERIFY-1 and AERIFY-2 itepekimab trials in COPD had mixed results reported May 2025; Mabwell's data support continued ST2 blockade testing in the disease.
Roivant Sciences (NASDAQ: ROIV) analyst commentary continued through the weekend following Tuesday's Pulmovant Phase 2 PHocus mosliciguat win, with Leerink Partners' David Risinger flagging up to $10 billion peak sales potential for mosliciguat across multiple pulmonary hypertension indications. The Leerink estimate substantially exceeds the ~$3 billion PH-ILD-only estimate Guggenheim disclosed Wednesday September 9. Other analyst peak sales projections ranged from $1.5 billion to $5 billion depending on indication assumptions and market-share methodology. The week's analyst upgrade sequence: Jefferies to $52 (from $42), Guggenheim to $46 (from $42), Piper Sandler to $55 (from $40), TD Cowen to $55 (from $50), BofA also raised. Consensus 12-month price target clusters around $50-55. The PHocus trial produced a 56.3% placebo-adjusted PVR reduction at Week 16 (p<0.0001), 6MWD +35.2 meters, and NT-proBNP −357.7 pg/mL (−53.2%). The Phase 3 PHrontier trial in approximately 375 patients is enrolling. Mosliciguat's mechanism (inhaled soluble guanylate cyclase activator working independently of heme and nitric oxide) differentiates it from sGC stimulators like Bayer's Adempas (riociguat) and prostacyclin analogs like United Therapeutics' Tyvaso (treprostinil).