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Weekly News Round Up - February 1, 2012

FDA Approves Kalydeco For Rare Form of Cystic Fibrosis

Kalydeco (ivacaftor) approved for CFTR gene mutation in patients six years of age and older Read More...

Cystic fibrosis (CF) is an inherited disease that results in faulty transport of salt (chloride) and fluids from the lungs and digestive tract that can lead to serious infections. Kalydeco (ivacaftor) has been FDA-approved for patients 6 and older that have the G551D mutation on the Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) gene. In the U.S., about 1,200 out of 30,000 CF patients have this faulty G551D mutation. Kalydeco improves the transport of salts and fluids in the body, reducing mucous build-up and leading to improved lung function and weight gain. Kalydeco improved lung function (FEV1) significantly in two 48-week, placebo-controlled studies. Kalydeco treatment for one patient will cost about $300,000 per year.

Bydureon: First Once-A-Week Treatment for Type 2 Diabetes Authorized

Bydureon (exenatide extended-release for injectable suspension) approved; boxed warning signals risk for thyroid cancer Read More...

Amylin Pharmaceuticals has received FDA-approval of Bydureon (exenatide extended-release for injectable suspension), a once-a-week, 2 mg injection used in conjunction with diet and exercise to improve glycemic control in type 2 diabetics. Exenatide is the same active ingredient in Byetta, which is given twice daily. The 24-week DURATION-5 clinical trial demonstrated that Bydureon statistically reduced A1C, a measure of blood sugar control, by 1.6 percentage points compared to a 0.9 percentage point reduction with Byetta. Nausea was the most frequently reported adverse event. A boxed warning of the occurrence of thyroid C-cell tumors at clinically relevant doses in rats accompanies the Bydureon approval; however, it is not known if Bydureon causes medullary thyroid carcinoma (MTC) in humans.

Picato Gel for Actinic Keratosis Available in March

Shorter treatment duration is an advantage for Picato (ingenol mebutate) Gel; available in two strengths Read More...

Actinic keratosis (AK) can be a precursor to squamous cell carcinoma, the second most common type of skin cancer. Picato (ingenol mebutate) Gel is now approved in a 0.015% strength for the face and scalp area to be used once daily for three consecutive days, or a 0.05% strength used once-daily for two consecutive days on the trunk and extremities. Picato is the first AK treatment with a short 2 or 3-day treatment duration. Placebo-controlled trials in over 1,000 patients with AK demonstrated significantly greater complete lesion clearance with Picato. The most common adverse effects were local skin reactions such as redness, flaking/scaling, crusting and swelling.

Inlyta Approved For Advanced Renal Cell Carcinoma

Inlyta (axitinib) represents seventh drug approval for metastatic kidney cell cancer since 2005 Read More...

Inlyta (axitinib) is an oral kinase inhibitor approved for the treatment of advanced renal cell carcinoma (kidney cancer) in adults after failure of one prior therapy. A single, randomized, open-label clinical trial (n=723) demonstrated that Inlyta resulted in a median progression-free survival of 6.7 months compared to 4.7 months with Nexavar (sorafenib), also a renal cell cancer drug. Common side effects include diarrhea, high blood pressure, and fatigue. High blood pressure should be well controlled in all patients prior to treatment. Avoid strong CYP3A4/5 inhibitors with Inlyta. Other approved agents for renal cell carcinoma include sunitinib, temsirolimus, everolimus, bevacizumab, and pazopanib.

Erivedge Receives Priority Approval For Metastatic Skin Cancer

Erivedge (vismodegib) treats locally advanced or metastatic basal cell cancer; oral, once-daily capsule approved Read More...

Basal cell carcinoma is the most common form of skin cancer due to sun damage. But when the cancer is no longer treatable by surgical or radiation measures, or the disease has spread in the body, approved treatments are limited. The U.S. Food and Drug Administration has approved Erivedge (vismodegib), the first hedgehog pathway inhibitor available for treatment of metastatic basal cell cancer.  In Genentech trials, a partial response was seen in 30 percent of patients with metastatic disease, while a partial or complete response was seen in 43 percent of patients with locally advanced disease. The median duration of response was 7.6 months. Common side effects include muscle spasms, hair loss, altered taste and weight loss. Fetal defects or death may occur with Erivedge exposure in pregnancy.

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