FDA expedited program (new guidance for draft) !!

FDA Grants Full Approval For PROMACTA® (eltrombopag) For Treatment Of Rare Blood Disorder

Article Date: 26 Feb 2011 - 1:00 PDT GlaxoSmithKline (NYSE: GSK) announced that the United States Food and Drug Administration (FDA) granted full approval for PROMACTA® (eltrombopag), an oral tablet that can raise platelet counts in patients with the rare blood disorder chronic immune (idiopathic) thrombocytopenic purpura (ITP) who have had an insufficient response to corticosteroids, immunoglobulins or splenectomy. PROMACTA initially received FDA orphan drug designation in May 2008 and accelerated approval in November 2008 for chronic ITP. The FDA Accelerated Approval program offers a pathway to gain provisional marketing approval for therapies that address unmet patient needs. Full approval of the therapy requires completion of post-marketing clinical trials and commitments that verify clinical benefit. "Full approval of PROMACTA was based on clinical studies that provide physicians and patients with a broader understanding of its treatment effect and safety profile," said Steven Stein, MD, V.P. of Medicines Development, GlaxoSmithKline. "PROMACTA is a testament to how the FDA Accelerated Approval Program supports development of therapies that meet unmet patient needs. Patients with limited treatment options gained access to PROMACTA while GSK conducted clinical studies that yielded additional efficacy and safety data."

FDA grants accelerated approval for GlaxoSmithKline, Genmab's Arzerra

October 27th, 2009 The FDA granted accelerated approval to GlaxoSmithKline and Genmab's Arzerra (ofatumumab) for use in patients with chronic lymphocytic leukaemia who have failed treatment with other therapies. The drugmakers noted that Arzerra is expected to be available in the coming weeks. The indication, which is for patients who are refractory to treatment with fludarabine and Genzyme's Campath (alemtuzumab), is based on results from a study in which 42 percent of patients with CLL responded to treatment with Arzerra. In May, an FDA advisory panel voted 10 to 3 that the data were "likely to predict clinical benefit" for the drug in this patient population. The FDA said that the approval requires further results from an ongoing study to confirm that addition of Arzerra to chemotherapy delays progression of the disease. According to an average estimate by analysts, Arzerra could reach sales of 282 million pounds ($462 million) by 2012.

Data supports fast-track approval of Roche's breast cancer drug, says FDA

11 September 2013 Faster approval of Roche's drug for early stage breast cancer, Perjeta, is supported by trial data, reviewers for the US Food and Drug Administration have announced this week. The Swiss pharmaceutical company is seeking approval of Perjeta as a neoadjuvant, which is a therapeutic agent administered before a main treatment, for patients whose cancer cells contain increased amounts of HER2 protein, but are still in the early stages of the disease. Perjeta, also known as pertuzumab, is already approved as a first-line treatment for metastatic breast cancer. For early stage breast cancer, the drug would be administered along with Roche's other cancer drug, Herceptin, and chemotherapy drug docetaxel. "FDA analysis of the Neosphere study showed statistically significant improvements in pCR rates by both the study and FDA-preferred definitions." If the drug is approved, it will become the first neoadjuvant breast cancer treatment approved by the FDA. The agency is trying to set up a new faster approvals process for early-stage breast cancer treatment. This includes a proposed new clinical goal known as pathologic complete response (pCR), which is defined as absence of invasive cancer in the breast. "FDA analysis of the Neosphere study showed statistically significant improvements in pCR rates by both the study and FDA-preferred definitions," FDA staff said, reports Reuters. Dietmar Berger, vice president of clinical development at Roche's Genentech unit, is reported by the news agency as saying that if Perjeta is considered in the neoadjuvant setting under the pCR pathway then it could be approved four years earlier than if the company were asked to wait for data from an ongoing trial not expected until 2016. An estimated 220,000 people are diagnosed with early breast cancer in the US each year.