Pfizer Announces FDA Approval of ELELYSO™ for Type 1 Gaucher Disease and Personal Support Program For Patients
On May 1, 2012, Pfizer announced the FDA approval of ELELYSO™ (taliglucerase alfa), an enzyme replacement therapy (ERT) for adults diagnosed with a rare genetic disorder called type 1 Gaucher disease. ELELYSO™, the first FDA-approved plant cell-based ERT for Gaucher disease, is also recognized for being acquired through a genetically engineered manufacturing system. This news [...]
FOR IMMEDIATE RELEASE – HR 5651, the Food and Drug Administration Reform Act of 2012
FOR IMMEDIATE RELEASE House FDA User Fee Bill Will Unlock Lifesaving Treatments for Rare Diseases Rare Disease Community Applauds House Energy & Commerce Committee for User Fee Reauthorization Legislation that Spurs the Development of Treatments Thursday, May 10, 2012, WASHINGTON, DC—– Today the House Energy & Commerce Committee finished mark-up of HR 5651, the “Food and [...]
Policy Update – PDUFA
We’re continuing to work on improving PDUFA to allow the FDA additional authority to include RARE disease in the Accelerated Approval Program. You can learn more here from last week’s public call for support and our other public policy blogs. After a very successful response from the patient community last week, today we are mobilizing organizations [...]
CALL TO ACTION !!
Rare Disease Language included in House draft PDUFA Bill RARE Project would like you to consider taking action in support of the new language (FAST Act) related to the Prescription Drug User Fee Act (PDUFA) in the following ways; The more support there is for the FAST Act, the better positioned the rare [...]
USA Today Spotlights FDA Approval
Advocating for your children can and does make a difference! On March 6, USA Today published an article spotlighting a brave family with twin girls suffering a rare and fatal genetic disease. Niemann-Pick Type C disease, often called childhood Alzheimer’s disease, has brought the Hempel family through rigorous challenges and constant battles in an effort to keep [...]
Change is Needed at the FDA
“Breakthrough technologies deserve a breakthrough in the way the FDA evaluates them.” - Andrew Von Eschenbach Mr. Von Eschenbach couldn’t have stated it any clearer. On February 14, the Wall Street Journal posted an on-line article by Mr. Eschenbach titled: Medical Innovation: How the U.S. Can Retain Its Lead, The FDA should approve drugs based on [...]
Christian Billingsley: “Waiting for Medical Science to Catch Up”
On February 7, 2012, WWL-TV, the CBS affiliate in New Orleans, reported on Christian Billingsley, a 17-year old boy diagnosed with atypical hemolytic uremic syndrome (aHUS) at 3 months of age. Online Story Just Keep Him Alive Until Medical Science Catches Up (click on title/link above to see WWL-TV video) Doug Mouton / Northshore [...]
RARE Disease Drug Development … it’s complicated!
The development of any drug is a very complicated process … for RARE diseases the task is especially difficult due to the limited patient populations. Typically the disease is less well understood and there are fewer patients to access for testing to prove the drug works. We’re hoping this simplified overview of the process will [...]
RARE DISEASE DAY EVENTS
Looking for a way to participate in Rare Disease Day Events? Don’t miss these Rare Disease Day events in DC! Monday, February 27, 2012 RDLA Luncheon Meeting & Conference Call (Host: Rare Disease Legislative Advocates) 12:30 – 2:00p.m. at 750 9th Street NW, Suite 750 Washington, DC 2nd Annual RDLA Cocktail Reception and [...]