The Food and Drug Administration has issued a positive review for a highly anticipated hepatitis C drug from Gilead Sciences, saying the pill cures more patients in less time than currently available treatments.
The agency posted its
review of Gilead's sofosbuvir online ahead of a meeting Friday where government experts will vote on whether to recommend the drug's approval.
More than 3 million people in the U.S. have hepatitis C, a blood-borne disease that causes liver damage and is blamed for 15,000 deaths a year. The drugs currently used to treat the virus cure about three-quarters of people and can take up to a year of treatment.
FDA said adding Gilead's sofosbuvir to the standard drug cocktail cured 90 percent of patients with the most common form of the virus in just 12 weeks.
The agency's reviewers state that the "shorter 12-week duration translates into a better tolerated side effect profile," adding that "no major safety issues associated with sofosbuvir have been identified to date."
Foster City, Calif.-based Gilead Sciences is one of a half-dozen drugmakers working to develop more effective treatments for hepatitis C, though many analysts predict the company's drug will eventually outperform its competitors. The FDA is expected to make a decision on the drug by Dec. 8.
Drugmakers see hepatitis treatments as a potentially lucrative market because the disease is expected to grow into a major public health problem as the baby boom generation ages. People born between 1945 and 1965 are five times more likely to have the virus than people of other age groups, and the Centers for Disease Control and Prevention is urging all baby boomers to get tested for the disease. Many Americans contracted the virus by sharing needles or having sex with an infected person in their youth.
For most of the last 20 years, the standard treatment for hepatitis C has involved a grueling one-year regimen of pills and injections that caused flu-like symptoms and cured less than half of patients. Then in 2011, the FDA approved two new drugs from Merck and Vertex Pharmaceuticals that raised the cure rate to about 65 and 75 percent, respectively, when combined with the older treatments.
Gilead's once-a-day pill appears to push the cure rate even higher.
In a company study of 327 patients with the most common form of the disease, 90 percent of participants had undetectable levels of the virus after 12 weeks of treatment. The form of the disease studied in the trial accounts for about 75 percent of hepatitis C cases in the U.S.
Gilead's drug was not as effective in treating two less common forms of the disease that account for about 25 percent of cases in the U.S. Among those patients, sofosbuvir cured about 67 percent of patients who had not previously taken other hepatitis C drugs.
But even for those patients, the FDA says Gilead's drug represents an important step forward.
The company's approach used only pill-based medications — sofosbuvir and another antiviral drug — while excluding interferon, the injectable medication that is the backbone of standard treatment, which can cause nausea, diarrhea and other unpleasant side effects.
For patients with the less common subtypes of the disease, Gilead's approach "provides the first all-oral, interferon-free treatment, as well as a shorter treatment duration and improved safety profile," according to the FDA's review.
Gilead is racing against other drugmakers to develop the first all-pill approach to treating the most common form of hepatitis C, long viewed as the holy grail of treatments by drugmakers. Similar efforts are underway from Abbott Laboratories, Bristol-Myers Squibb Co., Vertex Pharmaceuticals and others.
Pharmaceutical industry consulting firm Decision Resources estimates the market for hepatitis C drugs will grow to more than $23 billion by 2018. Sales of the drugs are expected to decline to $17.5 billion by 2021 as more patients are cured of the virus.
http://abcnews.go.com/Business/wireStory/fda-issues-positive-review-gileads-hep-drug-20655684
Gilead’s Hepatitis C Pill Works Better, FDA Report Says
By Anna Edney - Oct 23, 2013 9:50 AM ET
Gilead Sciences Inc. (GILD)’s experimental hepatitis C drug that lessens side effects and reduces length of treatment time is safe and effective against the disease, U.S. regulators said.
The medicine, sofosbuvir, has “a favorable benefit-risk assessment,” Food and Drug Administration staff said today in a review ahead of an Oct. 25 meeting of advisers to discuss the therapy. The FDA also will host an advisory panel tomorrow for
Johnson & Johnson (JNJ) and
Medivir AB (MVIRB)’s hepatitis C drug simeprevir.
Gilead, J&J, Medivir,
AbbVie Inc. (ABBV) and Bristol Myers Squibb Co. are among companies working on a new generation of hepatitis C drugs that alleviate the burden of current treatments that include interferon injections, which can cause flu-like symptoms. The total market for hepatitis C drugs may reach more than $100 billion over a decade, according to Bloomberg Industries.
For some patients, sofosbuvir “provides the first all-oral, interferon-free treatment, as well as a shorter treatment duration and improved safety profile compared to the current standard of care interferon-based regimen,” FDA staff wrote in the
report.
Sofosbuvir is estimated to generate $2 billion in sales next year, according to the average of eight analysts’ estimates compiled by Bloomberg.
Gilead rose 1.1 percent to $68.83 at 9:48 a.m.
New York time. The shares had gained 85 percent this year through yesterday.
Decision Date
The FDA didn’t find any heart risks associated with sofosbuvir after Bristol-Myers and Idenix Pharmaceuticals Inc. discontinued development of drugs in the same class last year based on cardiovascular safety concerns.
Gilead is seeking FDA approval of once-daily sofosbuvir combined for the majority of patients with pegylated interferon and another pill call ribavirin, both of which make up the backbone of current treatment for the virus as part of a regimen that can last as long as 48 weeks. Sofosbuvir can cut the treatment time to 12 weeks. J&J’s simeprevir gets therapy down to 24 weeks.
The FDA is scheduled to decide whether to approve sofosbuvir by Dec. 8, and simeprevir by Nov. 27.
Oral Combinations
For the majority of current patients interferon and ribavirin are combined with Merck & Co.’s Victrelis and
Vertex Pharmaceuticals Inc. (VRTX)’s Incivek. Victrelis, Incivek and J&J’s simeprevir are protease inhibitors that battle genotype 1 hepatitis C, the most common form of the disease. Sofosbuvir is the first in a new class of drugs called nucleotide polymerase inhibitors and is effective across all six hepatitis C genotypes.
About 4 million Americans have the disease, which can cause liver cirrhosis, according to the
National Institutes of Health. The disease can be passed through infected blood or body fluids, most commonly through needle-sharing by drug users. About 70 percent of U.S. hepatitis C patients carry the genotype 1 infection.
Gilead studied an all-oral combination of sofosbuvir and ribavirin for genotype 2 and genotype 3 patients and a combination with pegylated interferon and ribavirin for patients with the other genotypes, including 1, who haven’t been treated before.
Sofosbuvir combined with pegylated interferon and ribavirin for 12 weeks cured 90 percent of patients with genotypes 1,4,5 and 6 who hadn’t been treated before, Gilead said. FDA staff found data on patients with genotypes 5 and 6 to be insufficient to determine dosing because Gilead only studied 7 patients total with those types of hepatitis C.
New Data
“This is a two- or three-stage program,” John McHutchison, senior vice president of liver disease therapeutics at Gilead, said in an interview. “This is the first approval.”
Gilead is researching an all-oral combination of sofosbuvir and another Gilead drug ledipasvir the company hopes will hit the market about a year after sofosbuvir itself.
“Sofosbuvir seems to be the future of many of these highly effective all oral regimens,” McHutchison said.
FDA workers recommended yesterday potential simeprevir users be screened for a genetic mutation called Q80K polymorphism that renders the drug ineffective. No such suggestion was made regarding sofosbuvir.
Data from a mid-stage study on a combination of simeprevir and sofosbuvir with or without ribavirin are expected to be released in early November at the
American Association for the Study of Liver Diseases’ annual conference in Washington, D.C.
To contact the reporter on this story: Anna Edney in Washington at
aedney@bloomberg.net
To contact the editor responsible for this story: Reg Gale at
rgale5@bloomberg.net
http://www.bloomberg.com/news/2013-10-23/gilead-s-hepatitis-c-pill-works-better-fda-report-says.html
Preview - New wave of oral hepatitis C drugs near U.S. approval
By Deena Beasley
Wed Oct 23, 2013 10:08pm IST
(Reuters) - Patients infected with the liver-destroying hepatitis C virus should soon have better treatment options as new tablets from Gilead Sciences and others approach U.S. regulatory approval.
Gilead's experimental drug sofosbuvir is the first of a new wave of oral therapies to be considered by the Food and Drug Administration, which has convened an advisory panel for Friday.
The treatment is seen as the crown jewel of Gilead's $11 billion purchase of biotechnology company Pharmasset and aims to replace some of the pills and injections now on the market.
"Treatment will become simpler, shorter and safer ... a broader group of patients can be treated," said John McHutchison, head of liver disease therapeutics at Gilead.
In a pre-hearing report issued on Wednesday, FDA staff concluded that sofosbuvir is safe and effective as part of a combination regimen for hepatitis C.
The deadline for a regulatory decision on the drug is December 8. Wall Street analysts have forecast sales of $1.85 billion next year, assuming a price of $85,000 per patient, according to ISI Group. Hepatitis C affects around 3.2 million Americans, killing more than 15,000 each year, mostly from illnesses such as cirrhosis and liver cancer.
The standard treatment has long been a combination of interferon, given by injection, and ribavirin, a pill. But both drugs have side effects and neither works directly against the virus. In addition, the regimen is not that effective for many people living with hepatitis C, especially those with hard-to-treat genotype 1 infections.
Technological advances over the past decade have enabled the development of direct-acting antivirals (DAAs) that specifically target the hepatitis C virus. Two new antiviral drugs, Incivek from Vertex Pharmaceuticals Inc and Victrelis from Merck & Co, have cured more HCV patients in recent years, but both are protease inhibitors and are still used in combination with interferon and ribavirin.
Protease inhibitors are designed to block certain enzymes the virus needs to replicate.
Gilead's drug is part of a new class known as nucleotide analogue inhibitors, or "nukes," designed to block a different enzyme the virus needs to copy itself. The company says the therapy is more difficult for the body to overcome, and expects treatment times to be shorter and easier to endure. It has first sought FDA approval to use sofosbuvir along with ribavirin as an all-oral therapy for genotype 2 and 3 infections, or about 28 percent of the U.S. hepatitis C patient population.
It is also seeking approval to use the drug in combination with both ribavirin and interferon for more common genotype 1 infections, while planning for file next year for an all-oral treatment regimen for those patients.
"We have a very, very high barrier to resistance," McHutchison said.
He estimates that around 70 percent of U.S. HCV patients have genotype 1, up to 20 percent are infected with genotype 2, and 8 percent have genotype 3.
COMPETITIVE ADVANTAGE?
Gilead is the world's largest maker of branded drugs to treat HIV, the virus that causes AIDS, but has turned to hepatitis C treatments to diversify its pipeline.
"Before we acquired Pharmasset we did a lot of due diligence," McHutchison said. "I think we picked the right nuke."
Sanford Bernstein analyst Geoffrey Porges believes Gilead's advantage over competitors in the HCV field may be increasing as it becomes more clear that a nuke-based regimen has fewer limitations in terms of dosing convenience and breadth of genotype effectiveness.
"We are approaching an inflection point for hepatitis C for Gilead," he said.
It is a competitive field, and there have been setbacks for some. Vertex said in July that the FDA had placed a partial hold on a mid-stage study of its experimental hepatitis C nuke. Bristol-Myers Squibb last year discontinued development of one of its experimental drugs after a patient died of heart failure during a clinical trial and several others were hospitalized.
While Gilead is perceived to be in the driver's seat in the race to produce all-oral treatments, Bristol-Myers is expected to present Phase III data on an interferon-free treatment for genotype 1 patients at next month's meeting of the American Association for the Study of Liver Diseases in Washington D.C.
That regimen will consist of daclatasvir, which blocks a protein called NS5A that plays a key role in HCV replication, that Bristol-Myers sees as the backbone of its hepatitis program, along with its protease inhibitor asunaprevir.
Bristol plans to begin pivotal testing of a three-pill therapy early next year. Companies like AbbVie and Boehringer Ingelheim are also spending millions to develop new hepatitis C treatments.
McHutchison said data on another Gilead all-oral regimen - sofosbuvir combined with NS5A inhibitor ledipasvir - will be presented at a medical meeting next April. The company plans to file for FDA approval of that regimen, which includes genotype 1 patients, in the first half of next year.
He said sofosbuvir has so far been tested in more than 3,000 patients and there have been no issues with heart problems or other side effects beyond those associated with the drugs it has been combined with.
Earlier trials of the company's all-oral combination for genotype 1 resulted in more than 95 percent of patients being cured, McHutchison said.
The FDA advisory committee will also hear comment this week on Johnson & Johnson's application to sell simeprevir, an experimental protease inhibitor designed to target genotype 1 HCV.
(Additional reporting by Bill Berkrot; Editing by Krista Hughes)
(Reuters)
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