Rapamune, Pfizer's Branding-Troubled Drug, Gets FDA Approval for Rare Lung Disease

Rapamune, Pfizer’s Branding-Troubled Drug, Gets FDA Approval for Rare Lung Disease
May 29, 2015
By Mark Terry, BioSpace.com Breaking News Staff

Pfizer Inc. announced today that it had received the first ever approval by the U.S. Food and Drug Administration (FDA) for a treatment for lymphangioleiomyomatosis (LAM). LAM is a rare, progressive disease that affects the lungs, kidneys and lymphatic system.

Pfizer’s supplemental New Drug Application (sNDA) for the drug had been accepted by the FDA for priority review in February of this year. The drug already had approval in patients 13 years or older to prevent organ rejection in kidney transplant patients. In 2014 Rapamune generated global sales of $339 million.

LAM is very rare, with an incidence of two to five women per million worldwide having the disease. Abnormal smooth muscle cells invade lung tissues, airways and blood and lymph vessels, resulting in the destruction of the lung and difficulty in breathing. It is often fatal.

The clinical trial compared Rapamune in 89 patients compared to placebo over a 12-month period, followed by another year-long observation period. The primary endpoint was forced expiration volume—how much air the patient can exhale in one second. The difference compared to placebo was about 153 milliliters, but after discontinuation of the drug lung function returned to the same rate as patients on placebo.

“This approval is a landmark breakthrough for LAM patients to have access to this important treatment option,” said Susan Sherman, executive director of the LAM Foundation. “It is the result of decades of work by researchers and women of the LAM community who volunteered for this pivotal clinical trial.”

Several other clinical trials related to LAM are ongoing. The Cincinnati Children's Hospital Medical Center is currently enrolling a trial for women with LAM for a study using Rapamune (sirolimus). A study conducted by the is looking at the safety of simvastin in LAM. Symvastin is a statin drug most commonly used to treat cholesterol, manufactured by Merck & Co. .

Another trial is ongoing utilizing saracatinib. Saracatinib (AZD0530) is an experimental compound developed by AstraZeneca PLC that has been studied in Alzheimer’s disease and cancer.

In August 2014, Pfizer settled a class action lawsuit with 41 state attorneys general for alleged off-label marketing and promotion of Rapamune. Pfizer paid $35 million in the settlement. In the lawsuit, it was alleged that Wyeth, the company (bought by Pfizer) that developed the drug, misrepresented the drug’s uses and benefits.

This was a follow-up settlement to a 2013 settlement between Pfizer and the Department of Justice over Rapamune marketing. That settlement was worth $490.9 million. In that settlement, $257.4 million went to the federal government and states over alleged violations of the False Claims Act.

Pfizer also paid out $233.5 million for a criminal fine and forfeiture under a plea deal with the U.S. District Court in Oklahoma City as a result of Wyeth pleading guilty to a Federal Food, Drug and Cosmetic Act (FDCA) misbranding violation.

It was claimed that Wyeth trained the Rapamune sales force to push the drug’s use in non-renal transplant patients.

Will PfizerKline Become the Next Pharma Player?
The speculation surrounding a possible bid from Pfizer Inc. for struggling GlaxoSmithKline is heating up, after one closely-watched biotech analyst said in a note last week that Pfizer buying the company would “unlock access to its balance sheet and improve its tax situation.”

Gregg Gilbert, a biotech analyst at Deutsche Bank, wrote in a note to investors “Introducing PfizerKline” that he thinks a deal would be “materially accretive” for both companies. Gilbert estimated that a bid priced at $29.86 a share, via half stock and half cash, which would push up Pfizer’s earnings per share by 10 percent to 16 percent beginning in 2016.

“We believe that the company has a sense of urgency to create value by leveraging the power of its balance sheet to do needle-moving deals,” Gilbert wrote. “Since media reports in the past have pointed to the potential for a Pfizer/GSK combination, we are revisiting that theme.”

We want to know, dear readers, if you agree? Should Glaxo continue going it alone, or might Pfizer buy it and create one of the world’s largest pharma players in history?

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