In fiscal year 2011, FDA approved 7 “new molecular entities” (NMEs) in oncology.
6. Caprelsa (vandetanib) to treat thyroid cancer.
Importance: Caprelsa was the first drug approved to treat medullary thyroid cancer. There were previously no FDA-approved treatments for this type of cancer, a cancerous growth of the thyroid gland, which is located in the neck. Medullary thyroid cancer involves specific types of cells that are found in the thyroid gland and can occur spontaneously, or be part of a genetic syndrome. Medullary thyroid cancer is estimated to represent 3 to 5% of thyroid cancers, and is an orphan disease. It is estimated to affect about 1,300 to 2,200 patients in the U.S, making it one of the rarest forms of thyroid cancer. Caprelsa’s approval underscores FDA’s commitment to approving treatments for patients with rare and difficult to treat diseases.
Actions to speed drug testing and review: Caprelsa was given a Fast Track designation for its potential to treat a cancer with no approved treatments. Caprelsa’s safety and effectiveness were established in a single, randomized international trial of 331 patients with late stage medullary thyroid cancer. FDA did not require the sponsor to replicate the study findings in a second trial. Patients who received Caprelsa had a longer period of time without disease progression when compared to patients receiving placebo. Median progression-free survival was at least 22.6 months in the patients given vandetanib compared to 16.4 months in the patients given placebo. Caprelsa was reviewed under the FDA’s priority review program which provides for an expedited six-month review of drugs.
Safety issues: Caprelsa was shown to affect the electrical activity of the heart, which in some cases can cause irregular heart beats that could lead to sudden death. The drug was therefore approved with a REMS to inform health care professionals about these serious heart-related risks, without which the benefits of Caprelsa would not have been considered to outweigh the risks. Only health care professionals and pharmacies certified through the vandetanib REMS program, a restricted distribution program, will be able to prescribe and dispense the drug. Patients will also receive an FDA-approved Medication Guide informing them of the potential risks.
Source: FDA - FY 2011 Innovative Drug Approvals
Aucun commentaire:
Enregistrer un commentaire