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Rapamycin (Sirolimus) Compassionate Use and Expanded Access: How to Get It and Pay Less

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At a glance

  • Approved uses / Kidney transplant rejection prophylaxis and LAM [1]
  • Longevity status / Off-label; no approved aging or longevity use [1]
  • Approved forms / Rapamune tablets and oral solution; generic sirolimus tablets and solution [1]
  • Compassionate use / Reports in rare serious disorders, not healthy aging [3][4]
  • PEARL trial / 48 weeks; 5 mg or 10 mg compounded rapamycin once weekly [2]
  • Compounded status / Not FDA-approved; FDA does not verify safety, effectiveness or quality [5]

What is sirolimus approved for?

Rapamune (sirolimus) is labeled for prophylaxis of organ rejection in patients 13 years and older receiving renal transplants and for treatment of LAM [1]. The label covers Rapamune tablets and oral solution, and generic sirolimus tablets and oral solution are also approved [1]. There is no FDA-approved indication for aging, longevity or healthspan [1].

What does compassionate use mean for sirolimus?

In the sirolimus reports, compassionate use refers to treatment outside approved use for serious conditions, often after other therapies have failed [4]. One series described six children with complicated, life-threatening vascular anomalies who received sirolimus for compassionate use after failing multiple other therapies [4]. A review of RASopathy-associated hypertrophic cardiomyopathy described repurposing of sirolimus/everolimus, along with trametinib, on a compassionate-use basis [3]. These reports do not involve healthy aging.

Is there a research route for longevity?

The 48-week PEARL trial (NCT04488601) was a decentralized, double-blind, placebo-controlled randomized trial in healthy, normative-aging adults; participants received placebo, 5 mg or 10 mg compounded rapamycin once weekly [2]. Its primary outcome, visceral adiposity measured by DXA, did not change (p=0.942) [2]. Adverse and serious adverse events were similar across groups, and blood biomarkers remained within normal ranges [2]. The authors concluded it was relatively safe over 48 weeks and that efficacy was not established; no lifespan outcome was reported [2].

What does the label say to watch for?

The boxed warning is immunosuppression, and use is not recommended in liver or lung transplant patients [1]. Increased susceptibility to infection and possible development of lymphoma and other malignancies, particularly of the skin, may result from immunosuppression [1]. Other labeled risks include angioedema, fluid accumulation and impaired wound healing, hyperlipidemia, proteinuria, interstitial lung disease, male infertility, and fetal harm; the label recommends highly effective contraception during treatment and for 12 weeks after the last dose [1].

The label says to avoid strong inducers and strong inhibitors of CYP3A4 and P-glycoprotein [1]. It also states that grapefruit juice must not be taken with Rapamune or used to dilute it, and that cyclosporine increases sirolimus concentrations, with Rapamune taken 4 hours after cyclosporine [1].

Can sirolimus cost less?

Generic sirolimus tablets and oral solution are FDA-approved, while compounded rapamycin is not [1][5]. This page does not establish a discount or lower-cost route for longevity use. HealthRX.com offers rapamycin by prescription.

Frequently asked questions

Is rapamycin approved for longevity or compassionate use in aging?
No. Sirolimus is approved for kidney transplant rejection prophylaxis and LAM [1]. Published compassionate-use reports involve rare serious disorders such as complicated vascular anomalies and RASopathy-associated cardiomyopathy, not healthy aging [3][4].
Is compounded rapamycin FDA-approved?
No. Compounded drugs are not FDA-approved, and FDA does not verify their safety, effectiveness or quality before marketing [5]. The PEARL trial used compounded rapamycin [2].
What is the PEARL trial?
PEARL was a 48-week randomized, double-blind, placebo-controlled trial in healthy, normative-aging adults; participants received placebo, 5 mg or 10 mg compounded rapamycin once weekly [2]. Visceral adiposity did not change, adverse events were similar across groups, and no lifespan outcome was reported [2].

References

  1. U.S. National Library of Medicine, DailyMed. Rapamune sirolimus tablets and oral solution (Pfizer) prescribing information. Rapamune sirolimus tablets and oral solution (Pfizer) label
  2. Moel M, Harinath G, Lee V, Nyquist A, Morgan SL, Isman A, et al. Influence of rapamycin on safety and healthspan metrics after one year: PEARL trial results. Aging (Albany NY). 2025;17(4):908-936. Influence of rapamycin on safety and healthspan metrics after one year: PEARL trial results
  3. Chaput D, Andelfinger G. MEK Inhibition for RASopathy-Associated Hypertrophic Cardiomyopathy: Clinical Application of a Basic Concept. Can J Cardiol. 2024;40(5):789-799. MEK Inhibition for RASopathy-Associated Hypertrophic Cardiomyopathy: Clinical Application of a Basic Concept
  4. Hammill AM, Wentzel M, Gupta A, Nelson S, Lucky A, Elluru R, et al. Sirolimus for the treatment of complicated vascular anomalies in children. Pediatr Blood Cancer. 2011;57(6):1018-24. Sirolimus for the treatment of complicated vascular anomalies in children
  5. Compounding and the FDA: Questions and Answers | FDA. Compounding and the FDA: Questions and Answers | FDA
Evidence overview for Rapamycin (Sirolimus) Compassionate Use and Expanded Access: How to Get It and Pay Less