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NEWS

Relay Therapeutics’ Zovegalisib Receives the US FDA Breakthrough Therapy Designation for PIK3CA-Mutant Advanced Breast Cancer

Shots: The US FDA has granted BTD to zovegalisib + fulvestrant for the treatment of adults with PIK3CA mutant, HR+/HER2- locally advanced or metastatic breast cancer following recurrence or progression on or after treatment with a CDK4/6 inhibitor Designation was supported by P-I/II (ReDiscover) trial data of zovegalisib + fulvestrant ± CDK inhibitors, showing activity…
February 4, 2026

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Ipsen Pharmaceuticals
NEWS

Ipsen Receives US FDA Breakthrough Therapy Designation for IPN60340 in Unfit Acute Myeloid Leukemia

Shots: The US FDA has granted Breakthrough Therapy Designation (BTD) to Ipsen’s investigational therapy IPN60340 (ICT01) in combination with venetoclax and azacitidine for the treatment of 1L unfit acute myeloid leukemia (AML) The designation was supported by data from the P-I/II (EVICTION) study (n=57), where IPN60340 + Ven-Aza showed encouraging response rates, including a near…
January 14, 2026

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NEWS

Praxis Precision Medicines Reports the US FDA Breakthrough Therapy Designation of Ulixacaltamide for Essential Tremor

Shots: The US FDA has granted BTD to ulixacaltamide for the treatment of patients with essential tremor (ET) Designation was based on the topline data from the Essential3 program, consisting of Study 1 & 2, where in Study 1, ulixacaltamide improved mADL11 (1EP) by 4.3 points at Wk. 8, while Study 2 showed superior maintenance of…
December 30, 2025

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INSIGHTS+

New Drug Designations – September 2025    

Shots:     PharmaShots’ Designation Report provides a concise overview of the latest drug designations granted by major regulatory authorities, including the FDA, EMA, MHLW, Health Canada, and NMPA   The September 2025 report covers designations granted to 33 drugs and 4 medical devices, spanning 12 small molecules, 6 biologics, 8 cell and gene therapies & 4 medical devices among others  …
October 28, 2025

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NEWS

Daiichi Sankyo and Merck Report the P-II Study Data for Raludotatug Deruxtecan in Pt.-Resistant Ovarian, Primary Peritoneal, or Fallopian Tube Cancer

Shots: Daiichi Sankyo and Merck reported efficacy results for raludotatug deruxtecan (R-DXd) in patients with recurrent Pt.-resistant ovarian, primary peritoneal, or fallopian tube cancer from the P-II (dose-optimization) part of the REJOICE-Ovarian01 P-II/III study Across all doses (4.8 mg/kg, 5.6 mg/kg, and 6.4 mg/kg), ORR was 50.5% (n=107) as assessed by BICR, with 3 complete…
October 21, 2025

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NEWS

BeOne Medicines’ Sonrotoclax Receives FDA’s Breakthrough Therapy Designation for R/R Mantle Cell Lymphoma (MCL)

Shots: The US FDA has granted BTD to sonrotoclax for adults with r/r MCL & accepted BeOne’s request for participation in Project Orbis for concurrent global review of the therapy Decision was based on P-I/II (BGB-11417-201) assessing sonrotoclax in r/r MCL pts previously treated with BTKi & anti-CD20 therapy; Part 1 enrolled 22 pts on…
October 13, 2025

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Sanofi
NEWS

Sanofi Reports P-II (ALPHAMEDIX-02) Trial Data of AlphaMedix for Gastroenteropancreatic Neuroendocrine Tumors (GEP-NET)

Shots: Sanofi has reported data from the ongoing P-II (ALPHAMEDIX-02) trial assessing AlphaMedix (67.6μCi/kg, Q8W, ~4 cycles) in pts, incl. PRRT-naïve (n=35) & exposed (n=26) pts with histologically confirmed unresectable or metastatic SSTR+ GEP-NETs & ≥1 site of measurable disease Trial met all 1EPs with improved ORR & prolonged benefit, plus showed improvement across 2EPs…
October 8, 2025

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Ionis
NEWS

Ionis’ ION582 Receives the US FDA’s Breakthrough Therapy Designation to Treat Angelman Syndrome

Shots: The US FDA has granted BTD to ION582 (UBE3A protein expression stimulant) for the treatment of Angelman syndrome Designation was based on P-I/II (HALOS) trial assessing ION582 in pts with Angelman syndrome, which showed improvements across all functional domains incl. communication, cognition & motor function; safety was favorable Additionally, ION582 is being investigated in…
September 12, 2025

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Eli lilly
NEWS

The US FDA Grants Breakthrough Therapy Designation to Eli Lilly’s Olomorasib for KRAS G12C-Mutant NSCLC

Shots: The US FDA has granted BTD to olomorasib + Keytruda for the 1L treatment of pts with unresectable advanced or metastatic NSCLC harboring KRAS G12C mutation & PD-L1 expression ≥ 50% Designation was based on P-I/II (LOXO-RAS-20001) trial of olomorasib monotx. & dose optimization portion from P-III (SUNRAY-01) trial of olomorasib + Keytruda ±…
September 5, 2025

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