AstraZeneca社は、durvalumab（Imfinzi）とenfortumab vedotin併用による周術期治療の適応追加申請が、筋層浸潤性膀胱がん患者を対象に米国FDAで優先審査に指定されたと発表した。VOLGA第3相試験の中間解析で無イベント生存期間と全生存期間の有意な改善が示されたことが根拠となっている。

米国食品医薬品局（FDA）は、Insmed社の吸入抗菌薬アミカシンリポソーム吸入用懸濁液（ARIKAYCE）について、MAC（Mycobacterium avium complex）肺症を対象とした正式承認（フル承認）を求める一部変更承認申請（sNDA）を優先審査に指定し、審査完了目標日を2027年1月28日に設定した。

Carolyn Sheridan of NORD reviewed federal priorities (PDUFA reauthorization, PRV permanence, Scientific Expert Act) and state concerns (Medicaid work requirements and screening forms); John Hopper highlighted Rare Cancer Day and research needs for rare cancers.

Read a refresh of breast cancer FDA news from August 2026, including an sNDA application in PIK3CA-mutant disease and a fast track designation.

Jaguar Health (NASDAQ: JAGX) (“Jaguar”) is a commercial stage pharmaceuticals company focused on developing novel, plant-based, non-opioid, sustainably deri

Jaguar Health (JAGX) and family company Napo Pharmaceuticals received U.S. Food and Drug Administration waivers of 2027 fiscal year fees for two crofelemer-based products. Napo obtained a waiver of the Prescription Drug User Fee Act (PDUFA) fee for Mytesi (crofelemer delayed-release tablets), an FDA-approved prescription drug for symptomatic relief of noninfectious diarrhea in adult patients with HIV/AIDS on antiretroviral therapy, which is commercialized in the U.S. by Future Pak.Jaguar separately received a 2027 fiscal year fee waiver from the FDA’s Center for Veterinary Medicine for Canalevia-CA1, its conditionally approved prescription drug for treatment of chemotherapy-induced diarrhea in dogs, also commercialized in the U.S. by Future Pak. The announcement also reiterates the Jaguar group’s broader focus on plant-derived prescription drugs for gastrointestinal and mental health indications.

LEO Pharma has closed its acquisition of dersimelagon from Tanabe Pharma after the FDA accepted the drug's NDA for filing and granted Priority Review, with a PDUFA date expected by the end of February...

Scholar Rock faces a regulatory milestone for its drug candidate, apitegromab, as the FDA set a Prescription Drug User Fee Act (PDUFA) action date of September 30, 2026. The company is moving forward with the muscle-targeted therapy for spinal muscular atrophy (SMA) after addressing manufacturing co

Cytokinetics ($CYTK) Stock Hub updated Aug. 29, 2026: ACACIA-HCM and MAPLE-HCM analyses, MYQORZO launch, Nov. 14 PDUFA, cash and risks.

Axsome Therapeutics ($AXSM) Q2 2026: revenue $218.4M, net loss $51.3M, cash $319.9M. AUVELITY Alzheimer's agitation launch, AXS-12 PDUFA May 1 2027, pipeline, debt and insider activity. Not investment advice.

PHVS: CHAPTER-3 Q3 readout, September congress evidence, April 2027 PDUFA, cash, ownership and risk. Updated September 6, 2026.

What did the FDA's three-month extension add to the deramiocel BLA? 24-month HOPE-3 data, a narrower proposed indication, a November 22, 2026 PDUFA. $CAPR

Capricor Therapeutics (CAPR) will present 24‑month data from the Phase 3 HOPE-3 trial and its open-label extension of Deramiocel in Duchenne muscular dystrophy at the 2026 World Muscle Society Congress in Hiroshima, Japan.The late-breaking presentations will include skeletal muscle and cardiac outcomes, a delayed-start analysis and natural history comparisons that were included in a recent major amendment to the Deramiocel BLA, which has a PDUFA target action date of November 22, 2026. HOPE-3 randomized 106 patients; 82 reached 24 months (40 originally on Deramiocel, 42 on placebo). The trial met its primary endpoint, with Deramiocel slowing decline in upper limb function by 54% versus placebo on PUL 2.0 (p=0.03). Capricor will also present two preclinical posters from its StealthX exosome platform.

The FDA will meet Wednesday with patient groups and other stakeholders to discuss recommendations for reauthorizing the Prescription Drug User Fee Act. The agency is homing in on increasing regulatory transparency and proactive monitoring of chemistry, manufacturing and controls risk, while the public remains laser focused on rare disease. By Sept. 30, 2027, the U.S. Congress is expected to authorize the eighth iteration of PDUFA, which will set the FDA’s performance objectives, fee structures and program enhancements for fiscal years 2028-2032. Implemented by Congress in 1992, PDUFA allows the agency to collect fees from drug manufacturers to fund the review of new drug applications and must be reauthorized every five years. The user fees prescripted by PDUFA enable the FDA to complete drug review work but they by no means guarantee a favorable decision, Steven Grossman, president of policy and regulatory consulting firm HPS Group, told BioSpace. “It buys timely reviews and more FDA reviewers. It does not buy yes,” he said. Wednesday’s public meeting is expected to focus on proposed enhancements to pre-market drug review, post-market safety and chemistry, manufacturing and controls (CMC) risk, according to the public draft agenda—all hot button issues that continue to draw consternation among...

The FDA has set a PDUFA target action date of June 29, 2027, based on APEX trial data showing a 65% overall response rate in advanced systemic mastocytosis.

Phase 3 HAELO data showed an 87% reduction in monthly attack rates with Ionvoguran ziclumeran, with a PDUFA target date of March 10, 2027.

Teva has entered a stalking-horse bid for BXCL501, a sublingual agitation treatment under FDA review with a PDUFA date of 14 November 2026.