FDA gives green signal to new melanoma drug

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Swiss drugs manufacturer Roche Holding AG has captured a spectacular 30 percent of global drugs market. Photo - Pascal Lauener/Reuters

A new skin-cancer treatment that promises to shrink tumors and prolong the lives of patients with a particular genetic mutation won speedy approval. Forecasters believe the swift authorization signals U.S. regulators’ enthusiasm for such targeted therapies.

Vemurafenib, fromRoche HoldingAG andDaiichi SankyoCo., belongs to an emerging class of treatments that targets the molecular underpinnings of the cancer. These therapies offer the opportunity to significantly improve the treatment for patients who test positive for a genetic mutation that drives the proliferation of their cancer cells. Patients would take the drugs only after a test showed they have the mutation.

The U.S. Food and Drug Administration have approved what doctors are calling a revolutionary drug treatment for the deadliest form of skin cancer, and the pills are available right now.

The drug attacks a mutated gene found in about half of the cases of melanoma.The Roche manufactured drug called Zelboraf is the first treatment for melanoma that targets a specific gene found in skin-cancer tumors.

Melanoma is the fastest-growing form of cancer in terms of new diagnoses. Researchers attribute the acceleration to longer life expectancies and increased indoor tanning by young people.

The drug is sold as a pill, and patients are advised to take it twice a day.It is meant specifically for patients with melanoma tumors having what is called a BRAF genetic mutation.

Zelboraf- significant

“Zelboraf is a huge step forward in the fight against metastatic melanoma and the advancement of treatment for the deadliest form of skin cancer,” Dr. Anna Pavlick, director of the NYU Melanoma Program at the NYU Cancer Institute, New York City, said in a university statement. She took part in phase II and III trials of the drug and is an advisor to Zelboraf’s maker, Genentech.

“The approval of Zelboraf will make a significant impact on the survival and outcomes of advanced melanoma patients,” Pavlick added. “Patients taking the drug Zelboraf were 63 percent less likely to die from advanced melanoma than patients given standard chemotherapy alone,” she noted.

Another expert agreed that Zelboraf may be a potent weapon against melanoma for some patients.

The drug’s approval “represents a breakthrough accomplishment in targeting a therapy to a specific group of patients who can benefit,” Wendy K.D. Selig, president and chief executive officer of the Washington, D.C.-based Melanoma Research Alliance, said in a statement.

The treatment won’t cure the cancer entirely, but it will extend lives, doctors said.

“It’s only working in metastatic melanoma, or melanoma that spreads to different organ groups in the body,” said Dr. Timothy Jochen, a board certified dermatologist.

Jochen, with Contour Dermatology and Cosmetic Surgery Center in Palm Springs and Rancho Mirage, said almost half of all melanoma tumors have that mutation.He added data shows that the pills will extend life for about two months for most patients.

“People that had been on the drug for six months, 84 percent of those people were still alive with this drug, versus 64 percent on a traditional form of chemotherapy,” said Jochen.

Melanoma is a painful yet preventable disease.The disease can also be contracted by excessive use of tanning beds.

“Avoid peak hours of sun exposure. Wear sun-protective clothing, hats, and wear your sunscreen,” said Jochen, who also recommends that patients talk to any local dermatologist or oncologist for more information.

A six-month course of Zelboraf will cost about $56,400.

BRAF mutation

The FDA approval is specifically for patients with melanoma tumors that express a gene mutation called BRAF V600E.

BRAF typically helps regulate cell growth, but the variant of BRAF targeted by Zelboraf “is the abnormal gene mutation that allows melanoma cancer cells to spread,” Pavlick explained. “Since almost 50 percent of all melanoma tumors have the BRAF genetic mutation, this targeted drug therapy can be of great benefit to many patients.”

Along with the drug, the FDA also approved a companion test (made by Roche Molecular Systems) that will help determine if a patient’s melanoma cells have the BRAF V600E mutation. The first-of-a-kind test is called the cobas 4800 BRAF V600 Mutation Test.

“We now have the capability to analyze a patient’s melanoma tumor for the genetic mutation BRAF and use the targeted treatment Zelboraf to attack the tumor, shrink it and stop the progression of this deadly disease,” Pavlick said. “The drug comes in a simple pill form, taken twice a day.”

According to the FDA, Zelboraf’s approval came as part of an accelerated review program, and was based on a single international trial of 675 patients with late-stage melanoma with the BRAF V600E mutation who did not receive prior treatment. In the trial, patients received either Zelboraf or another cancer drug called dacarbazine. The median survival time after treatment was 8 months (64 percent still living) for patients in the dacarbazine group but has not been reached for patients in the Zelboraf group (77 percent still living), the agency said.

Still, not every person with melanoma will benefit from Zelboraf, and much more research is needed, the MRA’s Selig said.

“While the entire melanoma community applauds the breakthrough exemplified by vemurafenib [Zelboraf], the data demonstrate that tumors have the ability to develop resistance to the drug, causing patients to relapse,” she noted. “Additionally, because some patients with BRAF mutation do not respond to the drug and about half of patients do not have the mutation, finding additional new targets remains an ongoing urgent need.”

The most common side effects among patients taking Zelboraf were joint pain, rash, hair loss, fatigue, nausea and sun-related skin sensitivity. About 26 percent of the patients taking Zelboraf developed cutaneous squamous cell carcinoma, the second most common form of non melanoma skin cancer. It was managed with surgery.

Patients who take Zelboraf should avoid sun exposure, the FDA said.

Roche on top

Roche is already a major player in cancer treatment. Its three big therapiesAvastin, Herceptin and Rituxanaccount for 35% of its $47.8 billion total revenue and have helped drive its 30% share of the world-wide cancer market, according to Morningstar.

But Roche could use a new product with an estimated $1 billion in peak yearly sales. Tight budgets could force governments, including Medicare in the U.S., to limit the prices paid for cancer drugs.

According to the American Cancer society, over 70,000 Americans are diagnosed with melanoma each year, and nearly 9,000 will die from the disease annually.

In 2010, “8,700 people died of melanoma,” said Jochen. “So, it’s a very serious condition.”

Sources: Kesq, Usnews, Indystar

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