Chemistry:Garetosmab
Garetosmab, sold under the brand name Pasatru, is a fully human, IgG4 monoclonal antibody used for the treatment of fibrodysplasia ossificans progressiva. It is an activin signaling inhibitor against activin-A.[1]
The most common side effects include nosebleeds, increased hair growth, abscess, and acne.[2]
Garetosmab was approved for medical use in the United States in August 2026.[2]
Medical uses
Garetosmab is indicated to reduce formation of new heterotopic ossification lesions and clinician-assessed flare-ups in adults with fibrodysplasia ossificans progressiva.[1]
Fibrodysplasia ossificans progressiva is a rare genetic disease caused by a mutation in activin A receptor-type 1, which controls new bone growth.[2] As a result, connective tissues such as muscle, tendons and ligaments gradually turn into bone, causing limited movement, deformities, severe disability, and early death.[2]
Adverse effects
Garetosmab has a warning for fetal harm when administered during pregnancy.[2]
History
The effectiveness and safety of garetosmab were evaluated in a randomized, double-blind, placebo-controlled clinical study of 63 adults with fibrodysplasia ossificans progressiva.[2] Participants received garetosmab 3 mg/kg, garetosmab 10 mg/kg, or placebo, each given by intravenous infusion every 4 weeks for 56 weeks.[2] After the initial 56 weeks, 61 participants continued in an extended follow-up phase on the same treatment.[2]
Society and culture
Legal status
Garetosmab was approved for medical use in the United States in August 2026.[3] The US Food and Drug Administration (FDA) granted the application for garetosmab breakthrough therapy, fast track, orphan drug, and priority review designations.[2] The FDA granted the approval of Pasatru to Regeneron Pharmaceuticals.[2]
Names
Garetosmab is the international nonproprietary name.[4]
Garetosmab is sold under the brand name Pasatru.[2]
References
- ↑ 1.0 1.1 Cite error: Invalid
<ref>tag; no text was provided for refs namedPasatru FDA label - ↑ 2.00 2.01 2.02 2.03 2.04 2.05 2.06 2.07 2.08 2.09 2.10 "FDA Approves Treatment for Fibrodysplasia Ossificans Progressiva". U.S. Food and Drug Administration (FDA). 19 August 2026. https://www.fda.gov/drugs/news-events-human-drugs/fda-approves-second-treatment-fibrodysplasia-ossificans-progressiva.
This article incorporates text from this source, which is in the public domain.
- ↑ "Pasatru (garetosmab-grts) First and Only FDA-approved Treatment Demonstrating Reduction in New Heterotopic Ossification (HO) Lesions and Clinician-Assessed Flare-ups in a Placebo-controlled Trial in Adults with Fibrodysplasia Ossificans Progressiva (FOP)" (Press release). Regeneron Pharmaceuticals. 19 August 2026. Retrieved 18 September 2026 – via GlobeNewswire.
- ↑ "International nonproprietary names for pharmaceutical substances (INN): recommended INN: list 82". WHO Drug Information 33 (3). 2019.
Further reading
- Di Rocco, Maja; Forleo-Neto, Eduardo; Pignolo, Robert J.; Keen, Richard; Orcel, Philippe; Funck-Brentano, Thomas; Roux, Christian; Kolta, Sami et al. (October 2023). "Garetosmab in fibrodysplasia ossificans progressiva: a randomized, double-blind, placebo-controlled phase 2 trial". Nature Medicine 29 (10): 2615–2624. doi:10.1038/s41591-023-02561-8. PMID 37770652.
- Wang, Yuhuan; Nguyen, Jenny-Hoa; de Ruiter, Ruben R D; Mendell, Jeanne; Srinivasan, Dushyanth; Davis, John D; Eekhoff, Marelise W (October 2023). "THU467 Pharmacokinetics And Pharmacodynamics Of Garetosmab In Patients With Fibrodysplasia Ossificans Progressiva". Journal of the Endocrine Society 7 (Supplement_1). doi:10.1210/jendso/bvad114.428.
External links
- Clinical trial number NCT05394116 for "A Study to Assess Safety, Tolerability and Efficacy of Garetosmab Versus Placebo Administered Intravenously (IV) in Adult Participants With Fibrodysplasia Ossificans Progressiva (FOP) (OPTIMA)" at ClinicalTrials.gov
Template:Other drugs for disorders of the musculo-skeletal system
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