Great News for Thalassemia patients

Bluebird Bio confirms FDA approval of Zynteglo

bluebird bio (BLUE) announced the U.S. Food and Drug Administration has approved Zynteglo, also known as beti-cel, a one-time gene therapy custom-designed to treat the underlying genetic cause of beta-thalassemia in adult and pediatric patients who require regular red blood cell transfusions.

“Due to the complex nature of gene therapy, Zynteglo will be available exclusively at Qualified Treatment Centers, which are carefully selected based on their expertise in relevant areas such as stem cell transplantation, cell and gene therapy, and beta-thalassemia; and receive specialized training to administer Zynteglo.

Information on bluebird’s QTC network, as well as personalized support focused on the needs of each patient throughout their treatment journey and information on insurance coverage and access will be available through bluebird’s patient support program,” the company stated.

“The FDA approval of Zynteglo offers people with beta-thalassemia the possibility of freedom from burdensome regular red blood cell transfusions and iron chelation, and unlocks new possibilities in their daily lives.

Current thalassemia treatment

After more than a decade of research and clinical development, and through the perseverance of clinicians, patients, and their families, the approval of Zynteglo marks a watershed moment for the field of gene therapy.

As the first ex-vivo lentiviral vector gene therapy approved in the U.S. for the treatment of people with beta-thalassemia, we are ushering in a new era in which gene therapy has the potential to transform existing treatment paradigms for diseases that currently carry a lifelong burden of care,” said Andrew Obenshain, CEO of bluebird bio. 

Expensive Treatment

The lifetime cost of medical care for a patient with transfusion-dependent beta-thalassemia can reach up to $6.4M in the U.S. and the average total health care cost per patient per year is 23 times higher than the general population. bluebird estimates that there are approximately 1,300-1,500 individuals with transfusion-dependent beta-thalassemia in the U.S.

” bluebird has set the wholesale acquisition cost of Zynteglo in the U.S. at $2.8M, in “recognition of its robust and sustained clinical benefit demonstrated in clinical studies and its potential to alleviate a lifetime of health care costs associated with regular RBC transfusions and iron management.” Tom Klima, chief commercial and operating officer of bluebird bio, said.

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FDA Approved Vapes are coming!

FDA announces ‘first authorization’ for marketing of e-cigarette products

The U.S. Food and Drug Administration announced it has authorized the marketing of three new tobacco products, which it noted marks “the first set of electronic nicotine delivery system products ever to be authorized by the FDA through the Premarket Tobacco Product Application – PMTA – pathway.”

The FDA issued marketing granted orders to R.J. Reynolds Vapor Company, a subsidiary of British American Tobacco, for its Vuse Solo closed ENDS device and accompanying tobacco-flavored e-liquid pods, specifically, Vuse Solo Power Unit, Vuse Replacement Cartridge Original 4.8% G1, and Vuse Replacement Cartridge Original 4.8% G2.

“As the RJR Vapor Company submitted data to the FDA that demonstrated that marketing of these products is appropriate for the protection of public health, today’s authorization allows these products to be legally sold in the U.S.,” the FDA stated.

Solar Powered Vuse

Today, the FDA also issued 10 marketing denial orders for flavored ENDS products submitted under the Vuse Solo brand by RJR.

“Due to potential confidential commercial information issues, the FDA is not publicly disclosing the specific flavored products. These products subject to an MDO for a premarket application may not be introduced or delivered for introduction into interstate commerce. Should any of them already be on the market, they must be removed from the market or risk enforcement. Retailers should contact RJR with any questions about products in their inventory.

The agency is still evaluating the company’s application for menthol-flavored products under the Vuse Solo brand,” the FDA stated.

“Today’s authorizations are an important step toward ensuring all new tobacco products undergo the FDA’s robust, scientific premarket evaluation. The manufacturer’s data demonstrates its tobacco-flavored products could benefit addicted adult smokers who switch to these products – either completely or with a significant reduction in cigarette consumption – by reducing their exposure to harmful chemicals. We must remain vigilant with this authorization and we will monitor the marketing of the products, including whether the company fails to comply with any regulatory requirements or if credible evidence emerges of significant use by individuals who did not previously use a tobacco product, including youth. We will take action as appropriate, including withdrawing the authorization,” said Mitch Zeller, J.D., director of the FDA’s Center for Tobacco Products.

Mitch Zeller, J.D., director of the FDA’s Center for Tobacco Products

Shares to watch: BTI, MO, PM.

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