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Orphan Drugs: Start-Up LAM Therapeutics Gets FDA Designation

June 26, 2014

LAM Therapeutics, a Connecticut-based biopharmaceutical start-up company formed in April 2013, receives FDA Orphan Drug Designation (ODD) on June 25th for Sirolimus, for the treatment of the rare lung disease Lymphangioleiomyomatosis (LAM). The company has a disease-specific focus and singular mission to “advance the clinical development of new therapies to treat LAM.”

In June 2013, LAM Therapeutics announces that the company closed a Series A financing with private investors to launch operations to develop drugs for LAM. Proceeds from the Series A financing are used for the identification of clinical stage drugs with potential activity against LAM and to conduct clinical trials. The company has developed assays to identify clinical stage compounds having different mechanisms of action on a wide spectrum of targets with potential activity against LAM. LAM Therapeutics is the first biopharmaceutical company dedicated to identifying and developing drugs for the treatment of LAM.

FDA Orphan Drug Designation Database Record

Generic Name: Sirolimus
Trade Name: n/a
Date Designated: 06-25-2014
Orphan Designation: Treatment of Lymphangioleiomyomatosis (LAM)
Orphan Designation Status: Designated
FDA Orphan Approval Status: Not FDA Approved for Orphan Indication
Sponsor: LAM Therapeutics, Inc. 530 Old Whitfield Street Guilford, CT The sponsor address listed is the last reported by the sponsor to OOPD.


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: “Nessler Cylinders” by Panek (Own work) [GFDL or CC-BY-3.0 | via Wikimedia Commons.

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