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DTX401 (pariglasgene brecaparvovec), developed by Ultragenyx Pharmaceutical, shows a robust estimated probability of approval at 76%. This gene therapy targets glycogen storage disease type Ia, a rare metabolic disorder impacting around 6,000 patients in accessible markets, with a prevalence rate... Show more
Glycogen Storage Disease Type Ia (GSDIa)
The FDA has set a PDUFA action date for DTX401, which, if approved, will be the first treatment addressing the underlying cause of GSDIa, potentially improving patient quality of life.
MOLBREEVI (molgramostim) is an inhaled recombinant human granulocyte-macrophage colony-stimulating factor (rhGM-CSF) developed by Savara Inc. (SVRA) for the treatment of autoimmune pulmonary alveolar proteinosis (autoimmune PAP), a rare and debilitating lung disease characterized by pathological surfactant accumulation. The drug is... Show more
autoimmune pulmonary alveolar proteinosis (autoimmune PAP)
Drug: MOLBREEVI (molgramostim inhalation solution). Indication: autoimmune pulmonary alveolar proteinosis (autoimmune PAP). Timeline: August 22, 2026. FDA issued a Day 74 letter indicating no advisory committee is planned; the MOLBREEVI Biologics License Application (BLA) review is ongoing with a PDUFA... Show more
Deramiocel (CAP-1002), developed by Capricor Therapeutics, is an investigational allogeneic cardiosphere-derived cell therapy aimed at treating Duchenne muscular dystrophy (DMD). This therapy is positioned as a first-in-class treatment, targeting both skeletal muscle weakness and cardiac complications associated with DMD, addressing... Show more
Duchenne muscular dystrophy (DMD)
The PDUFA target action date for the Biologics License Application for Deramiocel is set for August 22, 2026, as the company works with the FDA toward potential approval.
Merck's KEYTRUDA (pembrolizumab), an anti-PD-1 monoclonal antibody, in combination with Astellas/Seagen's Padcev (enfortumab vedotin-ejfv), a Nectin-4-directed antibody-drug conjugate, represents a significant advancement in the treatment of advanced bladder cancer. This first-in-U.S. anti-PD-1 and ADC regimen has already transformed the treatment... Show more
muscle-invasive bladder cancer (MIBC) who are eligible for cisplatin-based chemotherapy
Drug: KEYTRUDA (pembrolizumab) in combination with Padcev (enfortumab vedotin-ejfv). Indication: muscle-invasive bladder cancer (MIBC) who are eligible for cisplatin-based chemotherapy. Timeline: 2026-08-17. FDA granted priority review for a supplemental BLA for KEYTRUDA combined with Padcev for cisplatin-eligible MIBC. The filing... Show more
KEYTRUDA QLEX (pembrolizumab and berahyaluronidase alfa-pmph), Merck's (MRK) subcutaneous formulation of the PD-1 inhibitor, is currently under FDA priority review for its use in combination with Padcev (enfortumab vedotin-ejfv), an antibody-drug conjugate (ADC) developed by Astellas and Seagen. This combination... Show more
muscle-invasive bladder cancer (MIBC) who are eligible for cisplatin-based chemotherapy
Drug: KEYTRUDA QLEX (pembrolizumab and berahyaluronidase alfa-pmph) in combination with Padcev (enfortumab vedotin-ejfv). Indication: muscle-invasive bladder cancer (MIBC) who are eligible for cisplatin-based chemotherapy. Timeline: 2026-08-17. FDA granted priority review for a supplemental BLA for KEYTRUDA QLEX combined with Padcev... Show more
Ziihera (zanidatamab-hrii), developed by Jazz Pharmaceuticals (JAZZ), is a bispecific HER2-directed antibody designed for dual epitope binding on HER2-positive tumor cells. This innovative mechanism promotes enhanced tumor growth inhibition, antibody-dependent cellular cytotoxicity (ADCC), and complement-dependent cytotoxicity (CDC) both in vitro... Show more
HER2-positive unresectable locally advanced or metastatic gastric, gastroesophageal junction (GEJ), or gastroesophageal adenocarcinoma (GEA)
Drug: Ziihera (zanidatamab-hrii). Indication: HER2-positive unresectable locally advanced or metastatic gastric, gastroesophageal junction (GEJ), or gastroesophageal adenocarcinoma (GEA). Timeline: August 25, 2026. FDA accepted Jazz's sBLA for Ziihera (zanidatamab-hrii) with Priority Review for combinations in first-line treatment of adult patients... Show more
LEQEMBI® IQLIK™ (lecanemab-irmb), developed by Eisai and Biogen (ticker: BIIB), signifies a significant advancement in the treatment of early Alzheimer's disease (AD). This best-in-class, amyloid beta-directed antibody aims to provide the same disease-modifying efficacy as the approved intravenous (IV) formulation... Show more
early Alzheimer’s disease (starting dose)
Drug: lecanemab-irmb (LEQEMBI IQLIK) subcutaneous injection. Indication: early Alzheimer’s disease (starting dose). Timeline: August 24, 2026. The FDA has extended review of the supplemental BLA for once-weekly lecanemab-irmb (LEQEMBI IQLIK) as a starting dose for early Alzheimer’s disease, designating the... Show more
Replimune Group (NASDAQ: REPL) is advancing its investigational therapy, RP1 (vusolimogene oderparepvec), a genetically modified herpes simplex virus type 1-based oncolytic immunotherapy, in combination with nivolumab for the treatment of advanced melanoma. This first-in-class agent is designed to locally destroy... Show more
advanced melanoma
Drug: RP1 (vusolimogene oderparepvec) in combination with nivolumab. Indication: advanced melanoma. Timeline: 2026-08-02. The FDA has accepted for review the resubmission of the BLA for RP1 combined with nivolumab for advanced melanoma and set a goal date for FDA action... Show more
mRNA-1010, developed by Moderna (MRNA), is currently in the NDA/BLA review stage, with a high estimated Probability of Approval (PoA) of 85.0%. This strong PoA is supported by robust immunogenicity data, acceptable safety profiles in over 14,000 adults, and alignment... Show more
seasonal flu vaccine
Drug: mRNA-1010. Indication: seasonal flu vaccine. Timeline: August 5, 2026. The U.S. FDA has assigned a Prescription Drug User Fee Act (PDUFA) goal date for mRNA-1010 of August 5, 2026.
MK-6240, developed by Lantheus Holdings (LNTH), is a first-in-class F18-labeled tau PET radiodiagnostic designed to detect neurofibrillary tangle (NFT) pathology in patients with cognitive impairment being evaluated for Alzheimer’s disease. Acquired by Lantheus in 2023 from Enigma Biomedical, MK-6240 targets... Show more
Detection of tau neurofibrillary tangle (NFT) pathology in patients with cognitive impairment being evaluated for Alzheimer’s disease
Drug: MK-6240. Indication: Detection of tau neurofibrillary tangle (NFT) pathology in patients with cognitive impairment being evaluated for Alzheimer’s disease. Timeline: August 13, 2026. The FDA has set a Prescription Drug User Fee Act (PDUFA) target action date of August... Show more
Iberdomide (BMS-986382), developed by Bristol Myers Squibb (BMY), is a cereblon E3 ligase modulator (CELMoD™) that operates by directly inducing tumor cell death and enhancing immune response through CRBN binding. This investigational drug is currently under evaluation for relapsed or... Show more
relapsed or refractory multiple myeloma (RRMM)
Drug: iberdomide. Indication: relapsed or refractory multiple myeloma (RRMM). Timeline: August 17, 2026. The FDA has granted a Prescription Drug User Fee Act (PDUFA) date of August 17, 2026 for iberdomide combined with daratumumab and dexamethasone (IberDd) in patients with... Show more
Ziihera (zanidatamab-hrii), developed by Zymeworks and now commercialized by Jazz Pharmaceuticals, is a bispecific HER2-targeted antibody designed to bind two distinct HER2 domains. This mechanism enables dual signaling blockade and enhances immune-mediated tumor cell destruction. Ziihera is currently under evaluation... Show more
first-line HER2-positive unresectable locally advanced or metastatic gastroesophageal adenocarcinoma (GEA)
Drug: Ziihera (zanidatamab-hrii). Indication: first-line HER2-positive unresectable locally advanced or metastatic gastroesophageal adenocarcinoma (GEA). Timeline: August 25, 2026. U.S. FDA accepted Jazz's sBLA for Ziihera (zanidatamab-hrii) for first-line HER2+ unresectable locally advanced or metastatic GEA and set a PDUFA target... Show more
Gilead Sciences (GILD) is advancing bictegravir 75 mg/lenacapavir 50 mg (BIC/LEN), an innovative single-tablet regimen (STR) for the treatment of HIV in adults who are virologically suppressed. This combination pairs bictegravir, a guideline-recommended integrase strand transfer inhibitor (INSTI) known for... Show more
treatment of HIV in adults who are virologically suppressed
Drug: bictegravir 75 mg/lenacapavir 50 mg (BIC/LEN). Indication: treatment of HIV in adults who are virologically suppressed. Timeline: 2026-08-27. FDA accepted the New Drug Application for bictegravir 75 mg/lenacapavir 50 mg (BIC/LEN) for the treatment of HIV in adults who... Show more
Gilead Sciences (GILD) is advancing its investigational once-daily single-tablet regimen, bictegravir 75 mg/lenacapavir 50 mg (BIC/LEN), aimed at treating virologically suppressed adults with HIV. This innovative combination pairs bictegravir, a guideline-recommended integrase strand transfer inhibitor (INSTI) known for its high... Show more
virologically suppressed people with HIV
Drug: BIC LEN. Indication: virologically suppressed people with HIV. Timeline: August 27, 2026. FDA accepted New Drug Application for bictegravir and lenacapavir (BIC LEN) for virologically suppressed people with HIV under priority review, with a PDUFA target action date of... Show more
ONS-5010/LYTENAVA™ (bevacizumab-vikg) is an ophthalmic formulation of bevacizumab being developed by Outlook Therapeutics (OTLK) for the treatment of neovascular age-related macular degeneration (nAMD), also known as wet AMD. As of the latest public information, the U.S. program is currently in... Show more
neovascular age-related macular degeneration (nAMD)
Drug: ONS-5010/LYTENAVA™ (bevacizumab-vikg). Indication: neovascular age-related macular degeneration (nAMD). Timeline: 2026-08-01. Outlook Therapeutics has resubmitted its Biologics License Application (BLA) for ONS-5010/LYTENAVA™ to the FDA. A PDUFA date and decision are expected within 60 days of the FDA's receipt of... Show more
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