Sirolimus is under clinical development by Pfizer and currently in Phase II for Amyotrophic Lateral Sclerosis. According to GlobalData, Phase II drugs for Amyotrophic Lateral Sclerosis have a 65% phase transition success rate (PTSR) indication benchmark for progressing into Phase III. GlobalData’s report assesses how Sirolimus’s drug-specific PTSR and Likelihood of Approval (LoA) scores compare to the indication benchmarks. Buy the report here.

GlobalData tracks drug-specific phase transition and likelihood of approval scores, in addition to indication benchmarks based off 18 years of historical drug development data. Attributes of the drug, company and its clinical trials play a fundamental role in drug-specific PTSR and likelihood of approval.

Sirolimus overview

Sirolimus (Rapamun, Rapamune, Rapalimus) is a macrolide lactams derivative drug belongs to immunosuppressive agent and an anti-neoplastic agent. Sirolimus is a macrocyclic lactone produced by Streptomyces hygroscopicus. It is formulated in the form of coated tablets, film coated tablets, tablets and solution for oral route of administration and gel for topical application. Rapamune is indicated for the prophylaxis of organ rejection in adult patients at low to moderate immunological risk receiving a renal transplant and also used to treat lymphangioleiomyomatosis (LAM), a rare, progressive lung disease that primarily affects women of childbearing age. Sirolimus is used for the treatment of lymphangioleiomyomatosis.

It is also under development for the treatment of primary immunodeficiency, lymphangioleiomyomatosis, vascular lymphatic anomalies, Pendred syndrome, cognitive impairment related to Sturge-Weber syndrome, epilepsy, myxoid liposarcoma, desmoid tumors (desmoid-type fibromatosis), chondrosarcoma, Castleman's disease, graft versus host disease (GVHD), amyotrophic lateral sclerosis, acute sensorineural hearing loss, growth disorders, ageing and angiofibroma. It was also under development for the treatment of graft versus host disease (GVHD), sudoriferous (Sweat) gland disorders, hemangioma and neurofibromatoses type I (Von Recklinghausen’s disease). It is administered orally as tablet and topically as a gel.

It was under development for the treatment of liver transplant rejection,  fibrodysplasia ossificans progressiva (myositis ossificans progressiva)

Pfizer overview

Pfizer discovers, develops, manufactures and commercializes biopharmaceuticals. The company offers products to treat various conditions such as cardiovascular, metabolic and pain, cancer, inflammation, immune disorders and rare diseases. It also provides sterile injectable pharmaceuticals, biosimilars, active pharmaceutical ingredients (APIs) and contract manufacturing services. Pfizer sells its products through wholesalers, retailers, hospitals, clinics, government agencies and pharmacies. It has major manufacturing facilities in India, China, Japan, Ireland, Italy, Belgium, Germany, Singapore, and the US. The company provides its products in North America, South America, Asia-Pacific, Australia, Europe, Africa and the Middle East. Pfizer is headquartered in New York, the US.

For a complete picture of Sirolimus’s drug-specific PTSR and LoA scores, buy the report here.


GlobalData, the leading provider of industry intelligence, provided the underlying data, research, and analysis used to produce this article.

GlobalData’s Likelihood of Approval analytics tool dynamically assesses and predicts how likely a drug will move to the next stage in its clinical pathway (PTSR), as well as how likely the drug will be approved (LoA). This is based on a proprietary algorithm built from the drugs’ sales forecast, regulatory milestones, cost forecasts, WACC rate and other proprietary data sources found on GlobalData’s Pharmaceutical Intelligence Center.