Biopharma · FDA Approvals

aflibercept · NCT07705555 registry update

countries: {'total_sites': 3, 'us_sites': 3} → {'total_sites': 6, 'us_sites': 6}

dabogratinib · NCT07265947 registry update

countries: {'total_sites': 25, 'us_sites': 19} → {'total_sites': 26, 'us_sites': 20}

brepocitinib · NCT05437263 registry update

status: ACTIVE_NOT_RECRUITING → COMPLETED; completion: 2026-07 → 2026-09-09

daraxonrasib · NCT07252232 registry update

countries: {'total_sites': 66, 'us_sites': 35} → {'total_sites': 74, 'us_sites': 40}

darovasertib · NCT07804186 registry update

countries: {'total_sites': 1, 'us_sites': 0} → {'total_sites': 13, 'us_sites': 11}

selinexor · NCT04756401 registry update

primary_completion: 2024-09-30 → 2025-08-29; enrollment: 52 → 8; primary_outcomes: ['Rate of minimal residual disease (MRD) negative status'] → ['Rate of Minimal Residual Disease (MRD) Negative Status']

povorcitinib · NCT06855498 registry update

countries: {'total_sites': 318, 'us_sites': 94} → {'total_sites': 297, 'us_sites': 87}

rezatapopt · NCT04585750 registry update

primary_completion: 2026-08-15 → 2026-12-15

Anfield Energy: Colorado DRMS recommends JD-8 mine permit approval

Colorado DRMS issued a completeness determination and staff recommendation for approval of the JD-8 mine permit. The Colorado Mined Land Reclamation Board will hold a formal public hearing on October 14-15, 2026 to consider the application.

Jazz Pharmaceuticals completes acquisition of Actio Biosciences for $820 million

Jazz Pharmaceuticals has completed its acquisition of Actio Biosciences for an upfront payment of $820 million. This acquisition adds ABS-1230, a clinical-stage therapy for KCNT1-related epilepsy, to Jazz's pipeline. The transaction aims to strengthen Jazz's epilepsy portfolio and its focus on rare diseases.

Summit presents updated HARMONi OS data at WCLC 2026

Updated June 2026 data cut shows sustained OS improvement with ivonescimab plus chemotherapy in the global HARMONi trial (HR 0.76, nominal p=0.0151) and consistent benefit in western patients (HR 0.76). Akeso also presented positive HARMONi-2 OS results showing ivonescimab monotherapy superiority over pembrolizumab (HR 0.73, p=0.009). No new safety signals were observed.

Vertex partners with Lindsey Vonn to promote JOURNAVX (suzetrigine)

The press release announces a marketing partnership with Lindsey Vonn to raise awareness of JOURNAVX. It confirms the FDA approval occurred on January 30, 2025 and states the product has been prescribed to over one million Americans. No new regulatory milestone or label change is disclosed.

Rocket Pharmaceuticals: FDA aligns on pivotal Phase 2 trial for RP-A501 in Danon disease

FDA confirmed the pivotal efficacy population of 12 male patients at the recalibrated dose of 3.8 × 10¹³ GC/kg, with the first three patients already treated counting toward this total. The agency also endorsed the 12-month co-primary endpoints of myocardial LAMP2 protein expression and a 10% reduction in left ventricular mass index, intended to support a potential accelerated approval pathway. Enrollment and dosing under the modified protocol may continue, with completion of dosing expected by mid-2027.

Rocket Pharmaceuticals: FDA aligns on 12-patient pivotal Phase 2 for RP-A501 in Danon disease

FDA confirmed the pivotal efficacy population of 12 patients, including the first three treated under the modified protocol, and endorsed the 12-month co-primary endpoints intended to support accelerated approval. The agency also cleared continued enrollment and dosing at the recalibrated 3.8 × 10¹³ GC/kg dose using commercial-grade product. Rocket now has a defined path to complete the pivotal study by mid-2027.

Vera Therapeutics: TRUTAKNA ORIGIN 3 final analysis shows eGFR stabilization, 76% kidney progression risk reduction

The ORIGIN 3 final efficacy analysis (n=428) met all prespecified endpoints, demonstrating true eGFR stabilization and a 76% reduction in composite kidney disease progression events through two years. Vera plans to submit a supplemental BLA for full approval in Q4 2026 based on these confirmatory results.

iBio September 2026 corporate presentation outlines obesity pipeline catalysts

The presentation states IBIO-610 IND-equivalent filing is expected in 2H 2026 and first patient dosing in 1H 2027; IBIO-600 interim Phase 1 data is anticipated in 1Q 2027; IBIO-800 IND-equivalent filing expected in 1H 2027.

Biofrontera: FDA approves Ameluz for superficial basal cell carcinoma

FDA approved the sNDA for Ameluz plus BF-RhodoLED red-light PDT for superficial basal cell carcinoma in adults. This makes Ameluz the first and only PDT approved in the US for a skin cancer and the only topical PDT indicated for both actinic keratosis and a skin cancer. The label expansion uses the same product and device already marketed for AK, enabling immediate commercial use.

Definium reports positive Phase 3 Panorama topline for DT120 ODT in GAD

Panorama met its primary endpoint and all key secondaries, showing a statistically significant 5.1-point placebo-adjusted HAM-A reduction at Week 12 (p<0.0001, d=0.64). This marks the third positive Phase 3 readout for DT120 ODT and the second in GAD, strengthening the regulatory package ahead of the planned pre-NDA meeting in 4Q 2026 and NDA filing in 1H 2027.

Scholar Rock: FDA approves ISEMBYLD (apitegromab-mstn) for SMA

FDA granted full approval for ISEMBYLD as the first muscle-targeted therapy for SMA in patients ≥2 years on SMN2 background therapy. The approval is supported by Phase 3 SAPPHIRE data showing statistically significant motor-function gains on the HFMSE scale. U.S. commercial launch is now underway with product shipping within days.

TELO: Telomir-Zn shows in-vitro anti-cancer activity in human/canine osteosarcoma

New Ohio State University data show Telomir-Zn inhibits growth in three osteosarcoma cell lines (HOS, D17, Abrams) at low concentrations. The company is now assessing an in-vivo canine osteosarcoma study alongside its ongoing TNBC Phase 1/2 program, opening a potential dual-pathway development strategy.