Alnylam Pharmaceuticals and Sanofi Report the US FDA’s Approval of Qfitlia (Fitusiran) to Treat Hemophilia A or B

Shots: The US FDA has approved Qfitlia to treat routine prophylaxis and prevent or reduce the frequency of bleeding episodes in pts. (age≥ 12yrs.) with hemophilia A or B, with or without factor VIII or IX inhibitors. Regulatory submissions have been completed in China & Brazil Qfitlia published clinical data in the NEJM in 2017, showing…

ByRidhi RastogiApril 1, 2025

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Genmab and Pfizer Secure the EC’s Approval for Tivdak (Tisotumab Vedotin) for Recurrent or Metastatic Cervical Cancer

Shots: The EC has approved Tivdak an ADC, as a monotx. for adults (n=502) with recurrent or metastatic cervical cancer with disease progression on or after systemic therapy Approval was backed by P-III (innovaTV 301) global trial evaluating Tivdak vs CT alone (topotecan, vinorelbine, gemcitabine, irinotecan, or pemetrexed) The trial met its 1EP of OS,…

ByDipanshu DixitApril 1, 2025

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AstraZeneca’s Calquence (Acalabrutinib) Plus Chemoimmunotherapy Receives the CHMP’s Positive Opinion for 1L Mantle Cell Lymphoma (MCL)

Shots: The CHMP has recommended Calquence + bendamustine & rituximab for the treatment of 1L MCL adults, who are ineligible for autologous HSCT; regulatory review is ongoing in Japan & other regions Opinion was based on P-III (ECHO) trial assessing the combination vs SoC (bendamustine & rituximab) in 1L MCL pts (≥65yrs.; n=635), which showed…

ByRidhi RastogiMarch 31, 2025

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Crinetics Pharmaceuticals Reports the EMA’s Acceptance of Paltusotine for Acromegaly

Shots: Following ODD grant, the EMA has accepted MAA of paltusotine for the treatment & long-term maintenance of acromegaly; NDA under the US FDA's review (PDUFA: Sep 25, 2025) Submission was supported by data from 18 trials, incl. 2 P-III trials (PATHFNDR-2 & PATHFNDR-1) assessing paltusotine vs PBO in treatment-naïve & experienced pts, respectively. These…

ByRidhi RastogiMarch 28, 2025

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Sanofi and Regeneron’s Dupixent Receives the MHLW’s Approval to Treat Chronic Obstructive Pulmonary Disease (COPD)

Shots: Japan’s MHLW has approved Dupixent to treat COPD in adults whose disease is inadequately controlled with existing therapy Approval was based on P-III (BOREAS) trial assessing Dupixent + SoC vs PBO in uncontrolled COPD adults with elevated blood eosinophils, which showed reduced exacerbations & improved lung function; data from BORUS & its replicate P-III (NOTUS)…

ByRidhi RastogiMarch 28, 2025

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Exelixis Reports the US FDA’s Approval of Cabometyx (Cabozantinib) to Treat Advanced Neuroendocrine Tumors (NETs)

Shots: The US FDA has approved Cabometyx for treatment-experienced, inoperable, LA/M, well-differentiated pancreatic NET (pNET) & extra-pancreatic NET (epNET) pts (≥12yrs.) Approval was based on P-III (CABINET) study assessing Cabometyx (60mg) vs PBO among 298 US pts (2 arms: pNET: n=99; epNET: n=199) that showed improved PFS; data was presented at ESMO 2024 & published…

ByRidhi RastogiMarch 27, 2025

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Sanofi Reports the US FDA’s Acceptance & Priority Review of Tolebrutinib for Non-Relapsing Secondary Progressive Multiple Sclerosis

Shots: The US FDA has accepted NDA & granted priority review to tolebrutinib for nrSPMS & slowing disability progression independent of relapse (PDUFA: Sep 28, 2025); MAA is under EMA review Submission was backed by P-III trials, incl. HERCULES for nrSPMS & GEMINI 1 & 2 for relapsing MS, along with clinical & preclinical data…

ByRidhi RastogiMarch 25, 2025

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Innovent Reports the NMPA Approval of Sycume for Thyroid Eye Disease

Shots: China’s NMPA has approved Sycume for the treatment of thyroid eye disease (TED) Approval was based on P-III (RESTORE-1) trial in TED pts, which met its 1EP in 2024, with 85.8% pts achieving ≥2mm proptosis reduction at 24wks., along with improved inflammation & QoL; data was presented at WOC, CSE, and CCOS Sycume (teprotumumab…

ByRidhi RastogiMarch 18, 2025

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Bayer Reports the US FDA’s sNDA Acceptance & Priority Review of Finerenone for Heart Failure (HF)

Shots: The US FDA has accepted sNDA & granted priority review to finerenone for HF pts having a left ventricular ejection fraction (LVEF) of ≥40%, i.e. mildly reduced (HFmrEF) or preserved LVEF (HFpEF), with approval expected in Q3’25. Awaits authorization in China, EU & Japan, with global applications planned Application was backed by P-III…

ByRidhi RastogiMarch 18, 2025

NEWS

AstraZeneca Reports the EC’s Approval of Imfinzi to Treat Limited-Stage Small Cell Lung Cancer (LS-SCLC)

Shots: The EC has approved Imfinzi monotx. for the treatment of LS-SCLC adults whose disease has not progressed after receiving Pt-based CRT; regulatory review underway in Japan & other regions Approval was based on P-III (ADRIATIC) trial (n=730) assessing Imfinzi (1500mg, IV, Q4W) with or without Imjudo (75mg, IV, Q4W × 4 cycles) followed by…

ByRidhi RastogiMarch 17, 2025

Alnylam Pharmaceuticals and Sanofi Report the US FDA’s Approval of Qfitlia (Fitusiran) to Treat Hemophilia A or B

Genmab and Pfizer Secure the EC’s Approval for Tivdak (Tisotumab Vedotin) for Recurrent or Metastatic Cervical Cancer

AstraZeneca’s Calquence (Acalabrutinib) Plus Chemoimmunotherapy Receives the CHMP’s Positive Opinion for 1L Mantle Cell Lymphoma (MCL)

Crinetics Pharmaceuticals Reports the EMA’s Acceptance of Paltusotine for Acromegaly

Sanofi and Regeneron’s Dupixent Receives the MHLW’s Approval to Treat Chronic Obstructive Pulmonary Disease (COPD)

Exelixis Reports the US FDA’s Approval of Cabometyx (Cabozantinib) to Treat Advanced Neuroendocrine Tumors (NETs)

Sanofi Reports the US FDA’s Acceptance & Priority Review of Tolebrutinib for Non-Relapsing Secondary Progressive Multiple Sclerosis

Innovent Reports the NMPA Approval of Sycume for Thyroid Eye Disease

Bayer Reports the US FDA’s sNDA Acceptance & Priority Review of Finerenone for Heart Failure (HF)

AstraZeneca Reports the EC’s Approval of Imfinzi to Treat Limited-Stage Small Cell Lung Cancer (LS-SCLC)

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