Monthly Archives: January 2019

Emmaus Life Sciences Launches Its Commercial Co-Payment Assistance Program for Endari™

TORRANCE, Calif., Jan. 28, 2019 /PRNewswire/ — Emmaus Life Sciences, Inc. (Emmaus), a leader in sickle cell disease treatment, announced today that it will provide financial assistance to help eligible patients afford their monthly co-payment for Endari™ (L-glutamine oral powder) [1] . The program is limited to financially eligible patients covered by commercial insurance.

A significant number of individuals with sickle cell disease are covered by commercial insurance. In some cases, patients may have difficulty affording the monthly co-payment amount.
Mark Diamond, Emmaus’ Vice President of Commercialization, commented: “We are committed to removing barriers between patients and Endari – giving a greater number of patients access to our effective treatment for sickle cell disease.”
Click here to read the full press release.
Sickle Cell Disease Association of America, Inc. (SCDAA) in no way endorses any medications, treatments, clinical trials, or studies reported through Get Connected. Information is provided to keep the readers informed. Because the manifestations and severity of sickle cell vary among individuals, personalized medical management is essential. Therefore, it is strongly recommended that all drugs and treatments be discussed with the reader’s physician(s) for proper evaluation and treatment.

 

CRISPR Therapeutics and Vertex Announce FDA Fast Track Designation for CTX001 for the Treatment of Sickle Cell Disease

CRISPR Therapeutics and Vertex Pharmaceuticals Incorporated today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track Designation for CTX001 for the treatment of sickle cell disease (SCD). CTX001 is an investigational, autologous, gene-edited hematopoietic stem cell therapy for patients suffering from severe hemoglobinopathies.

The FDA’s Fast Track program is designed to facilitate the development and expedite the review of drugs to treat serious conditions and fill an unmet medical need.  A drug granted Fast Track Designation may be eligible for several benefits, including more frequent meetings and communications with the FDA and, if relevant criteria are met, the potential for Accelerated Approval, Priority Review or Rolling Review of a Biologics License Application (BLA).

In October 2018, CRISPR and Vertex announced the FDA acceptance of the Investigational New Drug application (IND) for CTX001 for the treatment of SCD, and enrollment in a Phase 1/2 trial in SCD is currently underway in the U.S. The companies are also evaluating CTX001 for the treatment of β-thalassemia, and enrollment in a Phase 1/2 trial in β-thalassemia is currently open at multiple clinical trial sites in Europe.

Click here to read the press release issued today.