publication date: Jul. 7, 2017

Drugs and Targets

FDA clears cooling cap treatment in solid tumor chemotherapy

FDA cleared the expanded use of a cooling cap, DigniCap Cooling System, to reduce hair loss during chemotherapy.

This is the first cooling cap cleared by the agency for use in cancer patients with solid tumors.

Hair loss is a common side effect of certain types of chemotherapy and is commonly associated with the treatment of most solid tumor cancer. Hair may fall out entirely, gradually, in sections, or may become thin. Hair loss due to cancer treatment is usually temporary, but minimizing or relieving these kinds of side effects are considered important to overall treatment.

The DigniCap Cooling System is indicated to reduce the frequency and severity of hair loss during chemotherapy in solid tumor cancer patients in which alopecia-inducing chemotherapeutic agents and doses are used. It is a computer-controlled system used during treatment.

A cap is worn on the head and circulates liquid to a cap to cool the scalp during chemotherapy treatment. The cap is covered by a second cap made from neoprene, a type of rubber that holds the cooling cap in place and acts as an insulation cover to prevent loss of cooling.

The cooling is intended to constrict blood vessels in the scalp, which reduces the amount of chemotherapy that reaches cells in the hair follicles. The cold temperature also decreases the activity of the hair follicles and slows down cell division, making them less affected by chemotherapy. The combined actions are thought to reduce the effect chemotherapy has on the cells, which may reduce hair loss.

DigniCap may not work with some chemotherapy regimens, FDA said.

In 2015, the FDA granted marketing authorization of the DigniCap for use in patients with breast cancer. For that authorization, the efficacy of the cooling system was studied in 122 stage I and stage II women with breast cancer who were undergoing chemotherapy, using recognized chemotherapy regimens that have been associated with hair loss.

That study demonstrated that more than 66 percent of patients treated with the DigniCap reported losing less than half their hair. In support of the expanded use of the device, the manufacturer also submitted evidence from published, peer-reviewed articles that analyzed the application of the DigniCap to cancer patients with solid tumors in other areas of the body besides the breast. The FDA concluded that these studies provided valid scientific evidence to support the safety and efficacy of the expanded indication for the DigniCap.

The device is contraindicated for pediatric patients, patients with certain cancers and patient undergoing specific chemotherapy treatments. Additionally, DigniCap may not be appropriate for patients with cold sensitivity or susceptibility to cold-related injuries.

Long-term effects of scalp-cooling and risk of scalp metastasis have not been fully studied.


FDA granted marketing approval to the Praxis Extended RAS Panel

FDA granted marketing approval June 29 to the Praxis Extended RAS Panel, a next generation sequencing test to detect certain genetic mutations in RAS genes in tumor samples of patients with metastatic colorectal cancer.

The test is used to aid in the identification of patients who may be eligible for treatment with panitumumab (Vectibix, Amgen, Inc.).

This is the first FDA-approved NGS test that can detect multiple RAS gene mutations for colorectal cancer in a single test.

The Praxis Extended RAS Panel, sponsored by Illumina, Inc. detects the presence of 56 specific mutations in RAS genes (KRAS exons 2, 3, and 4 and NRAS exons 2, 3, and 4) in the tumor tissue of patients with mCRC.

If the Praxis Extended RAS Panel result indicates that a mutation is present in the colorectal cancer tissue, then panitumumab is not recommended. If the Praxis Extended RAS Panel does not detect a mutation—presumed to be RAS mutation-negative—then panitumumab may be an appropriate treatment.

The safety and effectiveness of the Praxis Extended RAS Panel was studied retrospectively using available specimens from mCRC patients enrolled in a multi-center clinical trial that evaluated panitumumab plus FOLFOX to FOLFOX alone.

Progression-free survival and overall survival in wild-type RAS patients demonstrated that the efficacy of panitumumab in patients whose tumors do not have a KRAS or NRAS mutation is supported. Analytical validation demonstrated that the panel performs consistently and accurately in the detection of the select KRAS and NRAS mutations in mCRC patients.

Panitumumab’s product labeling has been modified to align the indication for panitumumab and intended use for the Praxis Extended RAS Panel, stating that panitumumab is indicated in first-line therapy in combination with FOLFOX for patients with wild-type RAS—defined as wild-type in both KRAS and NRAS as determined by an FDA-approved test for this use—metastatic colorectal cancer.

Vectibix is also indicated as monotherapy for patients with wild-type RAS metastatic CRC following disease progression after prior treatment with fluoropyrimidine, oxaliplatin, and irinotecan-containing chemotherapy.


FDA approves Endari for patients with sickle cell disease

FDA approved Endari (L-glutamine oral powder) for patients age five years and older with sickle cell disease to reduce severe complications associated with the blood disorder.

“Endari is the first treatment approved for patients with sickle cell disease in almost 20 years,” said Richard Pazdur, acting director of the Office of Hematology and Oncology Products in the FDA’s Center for Drug Evaluation and Research and director of the FDA’s Oncology Center of Excellence. “Until now, only one other drug was approved for patients living with this serious, debilitating condition.”

FDA granted the approval of Endari to Emmaus Medical Inc.

Sickle cell disease is an inherited blood disorder in which the red blood cells are abnormally shaped, in a crescent, or “sickle,” shape. This restricts the flow in blood vessels and limits oxygen delivery to the body’s tissues, leading to severe pain and organ damage. According to NIH, approximately 100,000 people in the United States have sickle cell disease. The disease occurs most often in African-Americans, Latinos and other minority groups. The average life expectancy for patients with sickle cell disease in the U.S. is approximately 40 to 60 years.

The safety and efficacy of Endari were studied in a randomized trial of patients ages five to 58 years old with sickle cell disease who had two or more painful crises within the 12 months prior to enrollment in the trial. Patients were assigned randomly to treatment with Endari or placebo, and the effect of treatment was evaluated over 48 weeks.

Patients who were treated with Endari experienced fewer hospital visits for pain treated with a parenterally administered narcotic or ketorolac (sickle cell crises), on average, compared to patients who received a placebo (median 3 vs. median 4), fewer hospitalizations for sickle cell pain (median 2 vs. median 3), and fewer days in the hospital (median 6.5 days vs. median 11 days). Patients who received Endari also had fewer occurrences of acute chest syndrome (a life-threatening complication of sickle cell disease) compared with patients who received a placebo (8.6 percent vs. 23.1 percent).

Common side effects of Endari include constipation, nausea, headache, abdominal pain, cough, pain in the extremities, back pain and chest pain.

Endari received Orphan Drug designation for this use, which provides incentives to assist and encourage the development of drugs for rare diseases. In addition, development of this drug was in part supported by the FDA Orphan Products Grants Program, which provides grants for clinical studies on safety and/or effectiveness of products for use in rare diseases or conditions.

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