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Pfizer And Rare Lung Disease: FDA Approval Of Rapamune For Lymphangioleiomyomatosis (LAM)

May 28, 2015

The FDA announces late on May 28th, that Pfizer’s Rapamune (Sirolimus) is approved for the treatment of Lymphangioleiomyomatosis (LAM), a rare and progressive lung disease that affects mostly women of childbearing age. This is the first FDA approved drug for treating LAM.

Rapamune is available as both an oral solution and a tablet. Originally in 1999, Rapamune is approved as an immunosuppressive agent to help prevent organ rejection in patients 13 years and older receiving kidney transplants. According to the FDA Press Release, Rapamune received a Breakthrough Therapy Designation (BTD), priority review, and Orphan Drug Designation (ODD):

Generic Name: Sirolimus
Trade Name: Rapamune
Date Designated: 10-31-2012
Orphan Designation: Lymphangioleiomyomatosis (LAM)
Orphan Designation Status: Designated
FDA Orphan Approval Status: Not FDA Approved for Orphan Indication
Sponsor: Pfizer, Inc. 500 Arcola Road Collegeville, PA 19426


LAM is a rare lung disease found mostly in women that results in progressive cystic lung destruction. Besides the lungs, LAM can affect other organs including lymph nodes and kidneys. LAM is a “destructive, metastasizing neoplasm of smooth muscle-like cells that leads to progressive cystic lung disease. The disorder causes shortness of breath, lung destruction, respiratory failure, and death.” The disease originates from mutations in tuberous sclerosis complex (TSC) genes or can occur in otherwise healthy women (sporadic LAM). There is currently no cure or FDA-approved drug for LAM.

LAM Statistics

•   Approximately 200,000 LAM patients worldwide

•   At least 200 new cases of LAM diagnosed each year in the US

•   Average age of diagnosis is 35

•   After 10 years :

  1. 55% experience shortness of breath
  2. 20% require supplemental oxygen
  3. 10% die.


LAM Foundation

LAM Health Project.

Please Note: “Marty Schmidt in the Himalayas” by Sdiangelo (Own work) [CC-BY-SA-4.0]  | Wikimedia Commons.

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