A view shows the U.S. Food and Drug Administration (FDA) headquarters in Silver Spring, Maryland August 14, 2012. REUTERS/Jason Reed/File Photo

(Reuters) - The U.S. Food and Drug Administration on Monday approved Tesaro Inc’s drug, niraparib, for the treatment of recurrent ovarian cancer.

Epithelial ovarian, fallopian tube or primary peritoneal cancer affects the tissue covering the ovary or lining of the fallopian tube or abdominal wall. </s> REUTERS: The U.S. Food and Drug Administration on Monday approved Tesaro Inc's drug, niraparib, for the treatment of recurrent ovarian cancer.

Epithelial ovarian, fallopian tube or primary peritoneal cancer affects the tissue covering the ovary or lining of the fallopian tube or abdominal wall. </s> Shares of Dishman Pharmaceuticals jumped as much as 20 per cent to hit all-time high as the company has got FDA nod for cancer drug.

The company's partner Tesaro Inc has got U. Food and Drug Administration (FDA) nod for ovarian cancer drug Niraparib.

Niraparib kills cancer cells by inhibiting the production of proteins called PARPs, which help repair damaged DNA strands.

Research firm Dolat Capital says this drug has the potential to become very large given it is the first PARP inhibitor available as of now.

"We expect revenues from this molecule to Dishman can be anywhere between $40-80 million,'' Dolat Capital says.

More than 2.2 million shares traded, over twice their 30-day moving average.

Dishman stock was up 7.3 per cent this year as of Monday's close. </s> The U.S. Food and Drug Administration today approved Zejula (niraparib) for the maintenance treatment (intended to delay cancer growth) of adult patients with recurrent epithelial ovarian, fallopian tube or primary peritoneal cancer, whose tumors have completely or partially shrunk (complete or partial response, respectively) in response to platinum-based chemotherapy.

"Maintenance therapy is an important part of a cancer treatment regimen for patients who have responded positively to a primary treatment," said Richard Pazdur, M.D., 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. "Zejula offers patients a new treatment option that may help delay the future growth of these cancers, regardless of whether they have a specific genetic mutation."

Epithelial ovarian, fallopian tube or primary peritoneal cancer is a cancer of the tissue covering the ovary or lining the fallopian tube or abdominal wall (peritoneum). The National Cancer Institute estimates that more than 22,000 women will be diagnosed with these cancers in 2017 and more than 14,000 will die of these diseases.

Zejula is a poly ADP-ribose polymerase (PARP) inhibitor that blocks an enzyme involved in repairing damaged DNA. By blocking this enzyme, DNA inside the cancerous cells may be less likely to be repaired, leading to cell death and possibly a slow-down or stoppage of tumor growth.

The safety and efficacy of Zejula were studied in a randomized trial of 553 patients with recurrent epithelial ovarian, fallopian tube or primary peritoneal cancer, who had received at least two prior treatments of platinum-based chemotherapy and who had experienced a complete or partial response to their most recent chemotherapy treatment. Patients were tested with an FDA-approved test to determine whether they had a specific gene mutation, called a deleterious or germline BRCA mutation. The trial measured the length of time the patients' tumors did not grow after treatment (progression-free survival) in patients with and without the mutation. The median progression-free survival for patients taking Zejula who had a germline BRCA mutation was 21 months compared to 5.5 months for the same patient population taking a placebo. The median progression-free survival for patients taking Zejula who did not have a germline BRCA mutation was 9.3 months compared to 3.9 months for the same patient population taking a placebo.

Common side effects of Zejula include low levels of red blood cells (anemia), low levels of blood platelets (thrombocytopenia), low levels of white blood cells (neutropenia or leukopenia), heart palpitations, nausea, constipation, vomiting, abdominal pain/swelling (distention), inflammation of the mucous membranes (mucositis), diarrhea, indigestion (dyspepsia), dry mouth, fatigue, decreased appetite, urinary tract infection, liver problems (AST/ALT elevation), muscle pain (myalgia), back pain, joint pain (arthralgia), headache, dizziness, unusual taste sensation (dysgeusia), insomnia, anxiety, cold-like symptoms (nasopharyngitis), trouble breathing (dyspnea), cough, rash and high blood pressure (hypertension). Zejula is associated with serious risks, such as hypertension, severe increase in blood pressure (hypertensive crisis), bone marrow problems (myelodysplastic syndrome), a type of cancer of the blood called acute myeloid leukemia and low levels of blood cells in the bone marrow (bone marrow suppression).Women who are pregnant or breastfeeding should not take Zejula because it may cause harm to a developing fetus or a newborn baby.

The FDA granted this application Fast Track, Priority Review and Breakthrough Therapy designations.

Zejula also received Orphan Drug designation specifically for its use in treating recurrent epithelial ovarian cancer. This designation provides incentives to assist and encourage the development of drugs for rare diseases.

The FDA granted the approval of Zejula to Tesaro, Inc. </s> Feb 3 The U.S. Food and Drug Administration on Tuesday said it had approved Pfizer Inc's experimental breast cancer drug Ibrance, a regulatory decision that came more than two months earlier than expected.

Many analysts have considered the drug, whose chemical name is palbociclib, to be one of the most promising medicines in Pfizer's development pipeline, with potential for multibillion-dollar annual sales. (Reporting by Ransdell Pierson; Editing by David Gregorio) </s> Aug 30 (Reuters) - Novartis AG on Wednesday won highly anticipated U.S. approval for the first of a new type of potent gene-modifying immunotherapy for leukemia, a $475,000 treatment that marks the start of a potential new treatment paradigm for some cancers.

The approval was widely expected after an FDA advisory panel last month unanimously recommended the action, and Novartis shares closed virtually unchanged in Swiss trading on Wednesday.

Novartis announced an agreement with the U.S. Centers for Medicare and Medicaid Services under which payment for the therapy will be based on clinical outcomes achieved.

The treatment, to be sold under the name Kymriah, was approved for patients up to 25 years of age who have relapsed or were not helped by initial treatment for B-cell acute lymphoblastic leukemia (ALL).

Kymriah belongs to a new class of drugs called chimeric antigen receptor T-cell, or CAR-T therapies, that involves removing disease-fighting T cells from a patient, genetically modifying them to better recognize and attack cancer and then replacing them, where they can circulate for years seeking out the disease.

"We´re entering a new frontier in medical innovation with the ability to reprogram a patient´s own cells to attack a deadly cancer," U.S. Food and Drug Administration Commissioner Scott Gottlieb said in a statement.

U.S. biotech Gilead Sciences Inc jumped into the field this week with its announcement of an $11.9 billion deal to buy Kite Pharma. Gilead shares were up more than 5 percent on Wednesday as Kite is widely seen as likely to receive the next U.S. approval of a CAR-T therapy.

Shares of Bluebird Bio Inc, which is also developing a treatment in the emerging field, were up 11.8 percent at $114.47 on the Nasdaq.

"Two years ago many people would have told you these types of treatments were science fiction," said Brad Loncar, Chief Executive of Loncar Investments, which runs the Loncar Cancer Immunotherapy ETF

In clinical trials, CAR-T therapies have shown remarkable efficacy against blood cancers. In the pivotal Novartis trial, 83 percent of patients achieved remission with a disease that has historically poor outcomes.

"We've never seen anything like this before and I believe this therapy may become the new standard of care for this patient population," Dr Stephan Grupp of Children's Hospital of Philadelphia said in a statement.

However, this type of therapy is not without risk of severe side effects as the immune system goes into high gear. Kymriah will carry a boxed warning for cytokine release syndrome, a potentially lethal systemic response to the activation and proliferation of CAR-T cells, causing high fever and potential for neurological problems.

Juno Therapeutics Inc last year reported a handful of patient deaths during trials of its CAR-T therapy for leukemia. Its shares were down 5.7 percent at $41.30. (Reporting by Bill Berkrot and Mike Erman in New York, Natalie Grover in Bengaluru, Deena Beasley in Los Angeles and Julie Steenhuysen in Chicago; Editing by Shounak Dasgupta and Matthew Lewis) </s> Women with an aggressive type of breast cancer lived longer if they received immunotherapy plus chemotherapy, rather than chemo alone, a major study has found.

Women with an aggressive type of breast cancer lived longer if they received immunotherapy plus chemotherapy, rather than chemo alone, a major study has found.

The results are expected to change the standard of care for women like those in the clinical trial, who had advanced cases of “triple-negative” breast cancer. That form of the disease often resists standard therapies, and survival rates are poor. It is twice as common in African-American women as in white women, and more likely to occur in younger women.

Researchers said the new study was a long-awaited breakthrough for immunotherapy in breast cancer. Until now, most progress had been in other cancers, including lung cancer and melanoma, an aggressive skin cancer.

These findings may lead to the first approval by the Food and Drug Administration for an immunotherapy drug to treat breast cancer. But the approval would likely be limited to a certain type of aggressive cancer.

Although triple-negative tumors occur in only about 15 percent of patients with invasive breast cancer in the United States (or nearly 40,000 each year), they account for a disproportionate share of deaths, as many as 30 percent to 40 percent.

The term triple-negative refers to the tumors’ lack of sensitivity to the hormones estrogen and progesterone, and their lack of a protein called HER2, which is a target of treatment.

The immunotherapy in the study was atezolizumab (brand name Tecentriq), which belongs to a class of drugs called checkpoint inhibitors; the chemotherapy was nab-paclitaxel (Abraxane). Genentech is the maker of the immunotherapy drug in the study and paid for the research.

The findings were published Saturday in The New England Journal of Medicine, and were to be presented at a meeting of the European Society for Medical Oncology, in Munich. The study included 902 patients treated at 246 medical centers in 41 countries. Genentech, which is part of Roche, has already submitted the data to the FDA for approval.

Checkpoint inhibitors like atezolizumab work by helping T-cells — a type of white blood cell that is part of the immune system — recognize cancer and attack it. Research that led to these drugs won this year’s Nobel Prize in medicine.

The drugs generally work for fewer than half of patients but can bring lasting recoveries even to people who were severely ill. Side effects can be dangerous, even life-threatening, and treatment costs more than $100,000 a year.

In other cancers, researchers sometimes describe the tumors as “hot,” meaning they tend to have many mutations — genetic abnormalities that the immune system can recognize as foreign and attack.

But breast cancers tend to be relatively “cold,” with fewer mutations. The immune system is less likely to recognize them as invaders, which may help explain why previous studies of checkpoint inhibitors in breast cancer have been somewhat disappointing, researchers say.

In the new study, the key to success seems to have been giving chemotherapy along with immunotherapy.

“Chemo takes away the invisibility cloak the cancer has managed to put on,” Mayer said.

The chemo may help to ignite the immune system, in part by killing cancer cells that then spill substances the T-cells detect as foreign and begin to hunt.

The women in the study had triple-negative breast cancer that had been newly diagnosed and had become metastatic, meaning it had begun to spread. Once that occurs, the outlook is grim, with many patients surviving 18 months or less.

Half received chemo alone, and half were given chemo plus immunotherapy.

Among those who received the combination, the median survival was 21.3 months, compared with 17.6 months for those who received chemo alone. The difference was not statistically significant.

But when the researchers looked at women who had a marker called PD-L1 on their cancer cells, the results were striking: The median survival was 25 months in the combination group, versus 15.5 months with just chemo. That finding has not been analyzed statistically, and the patients are still being followed.

Doctors say the survival difference is important.

“This is truly a game changer,” said Dr. Sylvia Adams, an author of the study from NYU Langone Health’s Perlmutter Cancer Center.

Cancer patients with the PD-L1 marker tend to respond better to checkpoint inhibitors than those without it. In this study, 41 percent of patients had the marker. Genentech is seeking approval for treatment in triple-negative patients with the marker.

Adams said some patients, after initial treatment with both types of drug, have been doing well for two or three years with immunotherapy alone.

The “million-dollar question,” she said, is whether they can safely stop the immunotherapy if they have no sign of cancer.

She noted that patients in the study had some of the expected side effects of immunotherapy, including lung and pancreas inflammation.

Maribel Ramos, 42, was being treated at another hospital, which recommended chemo for her advanced triple-negative breast cancer.

“I was very worried because I know with that type of cancer, chemo doesn’t work,” Ramos said. She has three daughters: a 23-year-old and 10-year-old twins.

Her sister, a nurse at New York University, told her about the study there, and she began treatment in February 2016. She didn’t know it at the time, but she had been picked at random to receive the combined treatment. Within a few months, her tumors began to shrink. Nine months ago, for the first time, a scan found no sign of cancer. She is staying on immunotherapy.

“I just feel so happy that you can live longer,” Ramos said.

About 266,120 new cases of invasive breast cancer are expected in women in 2018 in the United States, and 40,920 deaths. </s> Oct 30 (Reuters) - Treatment of advanced squamous cell non-small cell lung cancer (NSCLC) with Bristol-Myers Squibb Co’s experimental immunotherapy nivolumab led to a one-year survival rate of 41 percent in a midstage clinical trial, according to data being presented at a medical meeting.

While the study called CheckMate-063 did not compare nivolumab with another drug or placebo, the historical one-year survival rate for patients like those in the trial, whose cancer had progressed after treatment with two or more prior therapies, is between 5.5 percent and 18 percent, the company said.

Nivolumab belongs to a highly promising new class of drugs called PD-1 inhibitors that block a tumor’s ability to camouflage itself, allowing the body’s immune system to recognize and attack the cancer. Merck & Co last month received the first U.S. approval of a drug from the class to treat advanced melanoma - the deadliest form of skin cancer.

“The Phase II findings from CheckMate-063 are encouraging as there are no effective treatment options for patients with refractory squamous cell lung cancer after their disease has progressed through two prior therapies,” Dr Suresh Ramalingam, director of Medical Oncology at Winship Cancer Institute of Emory University, said in a statement.

Bristol-Myers, which has proposed the brand name Opdivo for nivolumab, plans to use the data as part of its rolling submissions seeking U.S. and European approvals.

The median overall survival in the 117-patient trial was 8.2 months. The median duration of response had not yet been reached.

The objective response rate (ORR), which was the primary goal of the trial, was 15 percent as assessed by an independent review committee. That compares with historical expectations in the single digits, the company said. ORR was defined as reduction of the target lesion of at least 30 percent with no new lesions.

The results were scheduled to be reported on Friday at a thoracic oncology meeting in Chicago.

Fatigue, lung tissue inflammation and diarrhea were among the most common adverse side effects reported. The discontinuations rate due to drug-related side effects was 12 percent, and there were two drug-related deaths, the company reported.

Lung cancer is the leading cause of cancer deaths globally, with about 1.5 million each year, according to the World Health Organization. NSCLC accounts for about 85 percent of lung cancer cases. (Reporting by Bill Berkrot in New York; editing by Matthew Lewis) </s> Nov 24 The U.S. Food and Drug Administration said it had approved Eli Lilly & Co's Portrazza, in combination with two forms of chemotherapy, to treat patients with a type of lung cancer.

However, the treatment will need to carry a "boxed warning" that flags potential fatal risks, including cardiac arrest and sudden death. (1.usa.gov/1NbzJZ8)

A 1,093-patient clinical trial showed that patients who received Portrazza, along with chemotherapy drugs gemcitabine and cisplatin, survived an average of 11.5 months compared with 9.9 months for those who received only chemotherapy treatments.

The FDA's approval on Tuesday comes about four months after an advisory panel to the agency recommended the drug be cleared for sale along with the warnings.

Lung cancer is the leading cause of cancer death in the United States, with about 221,000 new diagnoses and 158,000 deaths so far in 2015.

Portrazza is approved to treat advanced squamous non-small cell lung cancer, the most common type of lung cancer.

It is not approved for the second type of non-small cell lung cancer, non-squamous cell.

Eli Lilly's shares were down 1.5 percent at $83.43 in early afternoon trading on the New York Stock Exchange. (Reporting by Ankur Banerjee and Rosmi Shaji in Bengaluru; Editing by Anil D'Silva and Ted Kerr)
The U.S. FDA approves Tesaro's ovarian cancer drug, Niraparib.