New Drugs 2013

Kadcyla for HER2-positive, late-stage (metastatic) breast cancer

Kadcyla (Ado-trastuzumab Emtansine) - Genentech - This drug reprents a new therapy for patients with HER2-positive, late-stage (metastatic) breast cancer. Kadcyla is intended for patients who were previously treated with trastuzumab, another anti-HER2 therapy, and taxanes, a class of chemotherapy drugs commonly used for the treatment of breast cancer. Kadcyla is trastuzumab connected to a drug called DM1 that interferes with cancer cell growth. Kadcyla delivers the drug to the cancer site to shrink the tumor, slow disease progression and prolong survival. It is the fourth approved drug that targets the HER2 protein. Results showed that patients treated with Kadcyla had a median progression-free survival of 9.6 months compared to 6.4 months in patients treated with lapatinib plus capecitabine. The median overall survival was 30.9 months in the Kadcyla group and 25.1 months in the lapatinib plus capecitabine group. The most common side effects reported were nausea, fatigue, pain in the muscles or joints, low levels of platelets in the blood (thrombocytopenia), increased levels of liver enzymes, headache, and constipation.

There is a BOXED WARNING that Kadcyla can cause liver toxicity, heart toxicity and death. The drug can also cause severe life-threatening birth defects, and pregnancy status should be verified prior to starting Kadcyla treatment.
Approved: Febuary 22, 2013

Kynamro for familial hypercholesterolemia

Kynamro (Mipomersen) - Genzyme - Approved for use in treating homozygeous familial hypercholesterolemia (HoFH) Kynamro must be given by injection once weekly. In one clinical trial, after 26 weeks of injections the average level of LDL-C "bad" cholesterol fell by 25%. Side effects were: flu-like symptoms, nausea, headache, and elevations in liver enzymes. This medication carries a BOXED WARNING of the possibility of serious liver toxicity.
Approved: January 29, 2013-02-20

Nesina for Type 2 diabetes

Nesina (Alogliptin) - Takeda - This drug was approved for the treatment of Type 2 diabetes. In extensive clinical studies Nesina lowered the HbA1c of 0.4% to 0.6% over 26 weeks of use. Common side effects were; runny nose, headache, and upper respiratory tract infections.
Approved: January 25, 2013 

Osphena for moderate to severe dyspareunia

Osphena (Ospemifene) - Shionogi - This drug has been approved for treating moderate to severe dyspareunia in postmenopausal women. Dyspareunia is associated with declining levels of estrogen during menopause, which leads to vulvovaginal atrophy, often resulting in pain during sexual intercourse. Ospemifene is a novel selective estrogen receptor modulator that makes vaginal tissue thicker and less fragile, resulting in a reduction in the amount of pain women experience with sexual intercourse. Ospemifene is taken orally with food once daily. The safety and effectiveness of ospemifene for dyspareunia were established in 1889 postmenopausal women with symptoms of vulvar and vaginal atrophy who were randomly assigned to receive ospemifene or a placebo. After 12 weeks of treatment, results showed a statistically significant improvement of dyspareunia in ospemifene-treated women compared with placebo-treated women. Common adverse effects of ospemifene reported during clinical trials included hot flushes/flashes, vaginal discharge, muscle spasms, genital discharge, and excessive sweating.

There is a BOXED WARNING - Ospemifene is being approved with a boxed warning alerting women and healthcare professionals that the drug, which acts like estrogen on vaginal tissues, has been shown to stimulate the endometrium and cause it to thicken. Women should see their physician if they experience any unusual bleeding as it may be a sign of endometrial cancer or a condition that can lead to it. It should be prescribed for the shortest duration consistent with treatment goals and risks for the individual woman, The boxed warning also states the incidence rates of thrombotic and hemorrhagic strokes (0.72 and 1.45 per 1000 women, respectively) and the incidence rate of deep vein thrombosis (1.45 per 1000 women).
Approved: February 26, 2013

Pomalyst for multiple myeloma

Pomalyst (Pomalid) - Celgene - Approved for the treatment of progressive multiple myeloma after treatment failure on other anti-cancer drugs. Pomalyst is a tablet that modulates the body's immune system to destroy cancerous cells and inhibit their growth. It is intended for patients who have received at least two prior therapies, including lenalidomide and bortezomib, and whose disease did not respond to treatment and progressed within 60 days of the last treatment (relapsed and refractory). Clinical results showed 7.4 percent of patients treated with Pomalyst alone achieved objective response rate (ORR). The median duration of response has not yet been reached in these patients. In patients treated with Pomalyst plus low-dose dexamethasone, 29.2 percent achieved ORR with a 7.4-month median duration of response.

Pomalyst carries a BOXED WARNING that the drug should not be used in pregnancy because it can cause severe life-threatening birth defects, and that the drug may cause blood clots.

Common side effects include a decrease in white blood cells (neutropenia), fatigue and weakness, low red blood cell count (anemia), constipation, diarrhea, low levels of platelets in the blood (thrombocytopenia), upper respiratory tract infections, back pain and fever.
Approved: February 8, 2013

Ravicti for urea cycle disorders

Ravicti (Glycerol Phenylbutyrate) - Hyperion - This is the 2nd drug approved for the treatment of Urea Cycle disorders (UCD's). The ability of this drug to control blood levels of ammonia was found comparable to the only other drug approved for this indication, sodium phenylbutrate (Buphenyl). UCDs are genetic disorders that involve deficiencies of specific enzymes involved in the urea cycle, a series of biochemical steps normally required to remove ammonia from the blood. When protein is absorbed and broken down by the body, it produces nitrogen as a waste product. The urea cycle removes nitrogen from the blood and converts it to urea, which is removed from the body through urine. In people with UCDs, nitrogen accumulates and remains in the body as ammonia, which can travel to the brain and cause brain damage, coma or death. Ravicti, a liquid taken three times a day with meals, helps dispose of ammonia in the body. It is intended for patients whose UCD cannot be managed by a protein-restricted diet or amino acid supplements alone. Ravicti must be used with a protein-restricted diet and, in some cases, dietary supplements. The most common side effects in patients treated with Ravicti include diarrhea, flatulence and headache.
Approved: February 1, 2013

 Uceris for Ulcerative Colitis

Uceris (Budesonide) - Santarus - This is a substantially improved method for delivering a steroid to the colon, the location of ulcerative colitis. Uceris, given in tablet form is taken once daily, does not release its steroid component until it reaches the colon. Clinical studies which compared Uceris with a placebo tablet resulted in 17-18% of patients experiencing a remission fo the colitis compared with 4-7% of patients treated with placebo. Side effects consited of nausea, decreased blood cortisol, upper abdominal pain, fatigue, abdominal distension, acne and urinary tract infections. It should be noted that the overall incidence of side effects did not differ from placebo.
Approved: January 15, 2013