22nd Aug 2011, 10:31 am by Brad Lemaire

Drug maker Vertex Pharmaceuticals (NASDAQ:VRTX) said Monday Health Canada has approved Incivek tablets for patients with chronic hepatitis C.

The drug, also known as telaprevir, is approved for use in combination with pegylated-interferon and ribavirin, two standard treatments used for Hep-C. Specifically, the drug can be used in patients with genotype 1 chronic hepatitis C, with compensated liver disease, meaning there is some damage to the liver, but the liver still functions.

About 170 million people worldwide are chronically infected with Hep-C, including some 250,000 Canadians. the serious liver disease can lead to liver damage, scarring, failure, or liver cancer.

The approval was based on three phase 3 studies of the drug of mroe than 2,500 people with hepatitis C. the results showed that people who took Incivek in combination with the other treatments achieved significantly higher rates of viral cure, compared to those that were treated with standard therapy alone.

The drug is indicated for people that are new to treatment, or did not respond to prior therapy.

“Health Canada’s rapid approval of Incivek underscores the urgent need for new medicines to treat hepatitis C,” said senior vice president and chief medical officer of Vertex, Robert Kauffman.

The company said that until recently, about 40% of people who underwent a year of treatment for Hep-C were cured.

“Incivek’s ability to nearly double that rate of cure and cut treatment time in half for the majority of patients being treated for the first time marks a turning point in the fight against this disease.”

Patients can live with the Hep C virus for many years without detecting any symptoms, leading for calls by some for increased hepatitis testing.

In 2010, the annual cost of hep-C due to medical treatment and lost productivity in Canada was estimated at $1 billion.

Vertex develops and commercializes small molecule drugs for the treatment of diseases. it is engaged in phase-one clinical trials for a host of drug candidates, which include drugs for the treatment for cystic fibrosis and influenza.

This past Friday, the U.S. Food and Drug Administration (FDA) approved the first of a new generation of chronic hepatitis C drugs, known as protease inhibitors. The drug, VICTRELIS? (boceprevir), from Merck & Co., has been shown to increase the rate of sustained viral response and shorten treatment times when used in combination with the current standard of care, ribavirin and pegylated alpha interferon, in the treatment of chronic hepatitis C. Vertex Pharmaceuticals is seeking approval to market a similar drug, INCIVEK? (telaprevir).

The addition of protease inhibitors to the current standard of care puts a new and significantly greater treatment burden on the patient. under most treatment regimens, pegylated alpha-interferon is injected once a week, and ribavirin is taken twice a day, for a total of five or six pills when prescribed in generic form. VICTRELIS is taken three times a day, for a total of 12 pills. INCIVEK is also taken three times a day, for a total of 6 pills. A treatment cycle lasting 48 weeks could mean that the patient is responsible for taking over 5,700 pills on schedule for the entire regimen. If the patient does not adhere to the prescribed regimen, the risk of treatment failure or relapse is increased (Reddy KR, Shiffman ML, Morgan TR, et al. Clin Gastroenterol Hepatol. 2007;5:124-129). Furthermore, because of the direct antiviral mechanism of protease inhibitors, missed doses of a protease inhibitor could lead to viral resistance (Weiss, et al. Aliment Pharmacol Ther 2009; 30:14-27).

Kadmon Pharmaceuticals’ proprietary RIBASPHERE? RIBAPAK? (ribavirin, USP) is the only ribavirin available in a daily, two-pill compliance package designed to enhance therapy adherence. with RIBASPHERE RIBAPAK, the patient takes only two pills each day — one in the morning and one at night — reducing the total ribavirin pill burden by up to 66 percent over a 48 week course of treatment. RIBASPHERE RIBAPAK packaging is clearly marked for seven days of AM and PM dosing, and the completion of a compliance pack reminds the patient to administer their accompanying weekly interferon therapy. Kadmon is also offering patients a daily therapy diary to help keep track of their treatment schedule.

“Maintaining treatment adherence under the burden of a triple therapy combination will require significantly greater vigilance from the patient,” said Bruce R. Bacon, M.D., professor of internal medicine at the Saint Louis University School of Medicine, and a clinical investigator for VICTRELIS. “With only one chance at success with this therapeutic approach, RIBASPHERE RIBAPAK represents an invaluable insurance policy for a treatment combination which could transform the enormous public health risks of hepatitis C.”

About Hepatitis C

Hepatitis C is a liver disease that results from infection with the hepatitis C virus. it can range in severity from a mild illness lasting a few weeks to a serious, lifelong illness. Hepatitis C virus can be either “acute” or “chronic.” Acute hepatitis C virus infection is a short-term illness that occurs within the first 6 months after someone is exposed to the hepatitis C virus. Seventy-five – 85% of acute HCV infections become chronic HCV infections. Chronic hepatitis C virus is a serious disease than can result in long-term health problems, such as serious liver disease, including cirrhosis and liver cancer, or even death. An estimated 3.2 million Americans have a chronic infection of the hepatitis C virus.

About RIBASPHERE? RIBAPAK? (ribavirin, USP)

INDICATION

RIBASPHERE? (ribavirin, USP) in combination with peginterferon alfa-2a is indicated for the treatment of adults with chronic hepatitis C virus infection who have compensated liver disease and have not been previously treated with interferon alpha.

IMPORTANT SAFETY INFORMATION

RIBASPHERE (ribavirin, USP) monotherapy is not effective for the treatment of chronic hepatitis C virus infection and should not be used alone for this indication (see WARNINGS). The primary clinical toxicity of ribavirin is hemolytic anemia. The anemia associated with ribavirin therapy may result in worsening of cardiac disease that has led to fatal and nonfatal myocardial infarctions. Patients with a history of significant or unstable cardiac disease should not be treated with ribavirin. Significant teratogenic and/or embryocidal effects have been demonstrated in all animal species exposed to ribavirin. In addition, ribavirin has a multiple dose half-life of 12 days, and it may persist in non-plasma compartments for as long as 6 months. Ribavirin therapy is contraindicated in women who are pregnant and in the male partners of women who are pregnant. Extreme care must be taken to avoid pregnancy during therapy and for 6 months after completion of therapy in both female patients and in female partners of male patients who are taking ribavirin therapy. At least two reliable forms of effective contraception must be utilized during treatment and during the 6 month post treatment follow-up period.

CONTRAINDICATIONS

RIBASPHERE (ribavirin, USP) is contraindicated in:

• Patients with known hypersensitivity to RIBASPHERE (ribavirin, USP) or to any component of the tablet.
• Women who are pregnant.
• Men whose female partners are pregnant, plan to become pregnant, or are not using contraception.
• Patients with hemoglobinopathies (e.g., thalassemia major or sickle-cell anemia).
• In combination with didanosine. Reports of fatal hepatic failure, as well as peripheral neuropathy, pancreatitis, and symptomatic hyperlactatemia/lactic acidosis have been reported in clinical trials.

RIBASPHERE (ribavirin, USP) and peginterferon alfa-2a combination therapy is contraindicated in patients with:

• Autoimmune hepatitis.
• Hepatic decompensation (Child-Pugh score greater than 6; class B and C) in cirrhotic CHC monoinfected patients before or during treatment.
• Hepatic decompensation with Child-Pugh score greater than or equal to 6 in cirrhotic CHC patients coinfected with HIV before or during treatment.

WARNINGS AND PRECAUTIONS

Treatment with RIBASPHERE (ribavirin, USP) and peginterferon alfa-2a should be administered under the guidance of a qualified physician and may lead to moderate to severe adverse experiences requiring dose reduction, temporary dose cessation or discontinuation of therapy.

RIBASPHERE (ribavirin, USP) must not be used alone because ribavirin monotherapy is not effective for the treatment of chronic hepatitis C virus infection.

RIBASPHERE (ribavirin, USP) and peginterferon alfa-2a should be discontinued in patients who develop evidence of hepatic decompensation during treatment.

There are significant adverse events caused by ribavirin/peginterferon alfa-2a therapy, including severe depression and suicidal ideation, hemolytic anemia, suppression of bone marrow function, autoimmune and infectious disorders, ophthalmologic disorders, cerebrovascular disorders, pulmonary dysfunction, colitis, pancreatitis, and diabetes. The peginterferon alfa-2a package insert and medication guide should be reviewed in their entirety prior to initiation of combination treatment for additional safety information.

Pregnancy: Ribavirin may cause birth defects and/or death of the exposed fetus. Extreme care must be taken to avoid pregnancy in female patients and in female partners of male patients.

Anemia: The primary toxicity of ribavirin is hemolytic anemia (hemoglobin < 10 g/dL), which was observed in approximately 13% of all ribavirin and peginterferon alfa-2a treated patients in clinical trials.

Hepatic Failure: Chronic hepatitis C (CHC) patients with cirrhosis may be at risk of hepatic decompensation and death when treated with alpha interferons, including peginterferon alfa-2a. Cirrhotic CHC patients coinfected with HIV receiving highly active antiretroviral therapy (HAART) and interferon alfa-2a with or without ribavirin appear to be at increased risk for the development of hepatic decompensation compared to patients not receiving HAART.

Hypersensitivity: Severe acute hypersensitivity reactions (e.g., urticaria, angioedema, bronchoconstriction, and anaphylaxis) have been observed during alpha interferon and ribavirin therapy.

Renal Impairment: RIBASPHERE (ribavirin, USP) should not be used in patients with creatinine clearance < 50 mL/min.

Pulmonary: Pulmonary symptoms, including dyspnea, pulmonary infiltrates, pneumonitis, pulmonary hypertension, pneumonia, and occasional cases of fatal pneumonia, have been reported during therapy with ribavirin and interferon. In addition, sarcoidosis or the exacerbation of sarcoidosis has been reported.

Bone Marrow Suppression: Pancytopenia (marked decreases in RBCs, neutrophils and platelets) and bone marrow suppression have been reported in the literature to occur within 3 to 7 weeks after the concomitant administration of pegylated interferon/ribavirin and azathioprine.

Pancreatitis: RIBASPHERE (ribavirin, USP) and peginterferon alfa-2a therapy should be suspended in patients with signs and symptoms of pancreatitis, and discontinued in patients with confirmed pancreatitis.

Laboratory Tests: Before beginning peginterferon alfa-2a/RIBASPHERE (ribavirin, USP) combination therapy, standard hematological and biochemical laboratory tests are recommended for all patients.

Drug Interactions: Nucleoside Analogues: NRTIs: In clinical trials, cases of hepatic decompensation (some fatal) were observed among the CHC/HIV coinfected cirrhotic patients receiving NRTIs. Patients receiving peginterferon alfa-2a/ribavirin and NRTIs should be closely monitored for treatment associated toxicities.

ADVERSE REACTIONS

Peginterferon alfa-2a in combination with ribavirin causes a broad variety of serious adverse reactions. The most common serious or life-threatening adverse reactions induced or aggravated by peginterferon alfa-2a and ribavirin include depression, suicide, relapse of drug abuse/overdose, and bacterial infections, each occurring at a frequency of < 1%. Hepatic decompensation occurred in 2% of CHC/HIV patients. nearly all patients in clinical trials experienced one or more adverse events.

For more information please see the accompanying RIBASPHERE RIBAPAK (ribavirin, USP) Tablets Full Prescribing Information. The peginterferon alfa-2a Package Insert should be reviewed in its entirety for additional safety information prior to initiation of combination treatment.

About Kadmon Pharmaceuticals

Kadmon Pharmaceuticals LLC is a privately held, new York City-based biopharmaceutical company founded on its expertise in novel science. The company explores new understandings in molecular biology to develop therapies that target the metabolomic or signaling pathways associated with disease, including novel anti hepatitis C therapies. Collaborating with academic centers and private enterprise at the forefront of innovation, Kadmon is focused on pioneering medicines in the areas of oncology, infectious diseases and immunology. for more information, visit kadmon.com.

The San Francisco Hepatitis C Task Force has called for a supervised safe injection facility for drug users in its final report.

The report, which came out in late January, also urged the city to do more to prevent and manage the life-threatening liver disease, including expanded testing and treatment.

Former Mayor Gavin Newsom established the task force in September 2009. the 30-member group includes public health officials, medical professionals, social service providers, treatment activists, and people living with hepatitis C.

“It’s great that Mayor Newsom and [current] Mayor [Ed Lee] have taken an interest in hepatitis C,” said task force member Dr. Brad Hare, medical director of the Positive Health Program at San Francisco General Hospital. “It is a major health concern for our city.”

The Centers for Disease Control and Prevention estimates that 3.2 million people in the U.S. have chronic hepatitis C, including approximately 12,000 in San Francisco. but because hepatitis C typically causes mild or no symptoms during its early stages, many people do not learn they are infected until they develop liver cirrhosis or cancer years later.

Hepatitis C virus is most often transmitted through blood-to-blood contact, for example sharing needles to inject drugs or hormones, or reusing piercing or tattooing equipment. Outbreaks of sexually transmitted hepatitis C among HIV-positive gay and bisexual men have been reported in several cities in the U.S. and Europe. but many people have no apparent risk factors and do not know how they became infected.

The task force report, “Recommendations for Strategically Addressing Hepatitis C in San Francisco,” is the outcome of a yearlong process to identify gaps in how the city responds to the disease. the full report is available online at hepcsf.org.

Among the key recommendations are establishing a hepatitis C coordinator within the Department of Public Health, starting a community planning council, making hepatitis testing and treatment more widely available, ensuring full access to hepatitis C care through the Healthy San Francisco program, and developing education and awareness campaigns.

“Our report highlights significant gaps in services for people like me who are living with hepatitis C and others at risk of infection,” said task force co-chair Dominique Leslie. “We urge Mayor Lee and the Board of Supervisors to take swift action to implement our recommendations.”

Although the recommendations come at a time when the city budget is stretched and services are being cut, the task force argues that spending money on hepatitis C prevention, testing, and timely treatment will pay off down the road.

“San Francisco has a unique opportunity to prevent new infections and save money for the city, provide care and treatment for those living with hepatitis C, prevent long-term complications, and be a leader in the national fight against this disease,” said task force member Dr. Todd Frederick of California Pacific Medical Center.

The report’s most controversial recommendation is creation of a pilot supervised safe injection facility, where drug users can administer drugs using clean needles under the watchful eye of trained staff. currently Vancouver has the only facility of this type in North America, which has been credited with lowering incidence of blood-borne infections and reducing overdose deaths.

“This particular recommendation is pushing the envelope,” Hare told the Bay Area Reporter, “but it really has an opportunity to improve the health of city residents who do inject.”

The task force will continue as a community coalition, advocating for full implementation of the report’s recommendations and continuing to educate public officials, medical and social service providers, high-risk groups, and the general public about hepatitis C and its impact.

The task force is currently seeking new members, focusing on San Francisco residents who are living with hepatitis C or belong to under-represented and at-risk groups including youth, people of color, transgender individuals, and drug users. Meetings are held the second Monday of each month and are open to the public. the application deadline is February 28. For more information contact .

U.S. FDA and Health Canada Grant Priority Reviews for Telaprevir for the Treatment of Hepatitis C -Six-month review date of May 23, 2011 set by FDA-

CAMBRIDGE, Mass.–(BUSINESS WIRE)–Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) announced today that the U.S. Food and Drug Administration (FDA) has accepted the new Drug Application (NDA) for telaprevir and granted the company’s request for six-month Priority Review. Telaprevir is Vertex’s lead medicine in development for people with genotype 1 chronic hepatitis C. the FDA grants Priority Review to medicines that offer major advances in treatment or provide a treatment where no adequate therapy exists. a target review date of May 23, 2011 is set under the Prescription Drug User Fee Act (PDUFA) for the FDA’s approval decision, which is four months earlier than the standard review time of 10 months.

Additionally, Vertex today announced the completion of a new Drug Submission (NDS) to the Therapeutic Product Directorate (TPD) of Health Canada seeking approval for telaprevir in Canada. Telaprevir was also granted Priority Review in Canada, which allows for faster review for promising medicines that address life-threatening or severely debilitating conditions and for which there are few effective therapies already available. Standard review in Canada takes 18 months or more and Priority Review typically shortens the review time to approximately six to nine months.

In December 2010, Janssen-Cilag International NV announced that the European Medicines Agency (EMA) accepted telaprevir for accelerated assessment in Europe, which is granted to new medicines of major public health interest.

“Data from Phase 3 studies showed that when compared to currently available medicines, telaprevir-based combination therapy nearly doubled viral cure rates and cut treatment time in half for the majority of patients new to treatment,” said Peter Mueller, Ph.D., Chief Scientific Officer and Executive Vice President of Global Research and Development at Vertex. “We look forward to working with the FDA and Health Canada to make telaprevir available as quickly as possible for people with hepatitis C.”

Data to Support the Telaprevir Submissions

The regulatory submissions in the United States, Canada and Europe are supported by data from three Phase 3 studies, known as ADVANCE, ILLUMINATE and REALIZE, which evaluated up to 12 weeks of telaprevir in combination with Pegasys® (pegylated-interferon alfa-2a) and Copegus® (ribavirin) in people chronically infected with genotype 1 hepatitis C virus (HCV) who were new to treatment as well as those who were treated before with currently available medicines but did not achieve a sustained viral response (SVR, or viral cure). in these studies, treatment with telaprevir-based combination therapy resulted in significantly higher viral cure rates compared to approved medicines, regardless of prior treatment experience, race or stage of liver disease. up to 75 percent of people new to treatment achieved a viral cure with telaprevir-based therapy. the majority of these people were able to complete their course of treatment at six months – half the time needed with currently available medicines. Among those who did not achieve a viral cure with a prior treatment course of currently available medicines, Phase 3 data showed that telaprevir-based combination therapy resulted in viral cure rates three to five times higher compared to re-treatment with currently available medicines. the safety and tolerability results of telaprevir-based combination therapy were consistent across the Phase 3 studies. the most common adverse events regardless of treatment regimen were rash, fatigue, pruritis, headache, nausea, anemia, insomnia, diarrhea, flu-like symptoms and pyrexia, with the majority being mild or moderate in severity.

Vertex provided a summary of Phase 3 results, including SVR and safety data for telaprevir, in its November 23, 2010 press release announcing the NDA submission.

Telaprevir is an investigational, oral inhibitor that acts directly on the HCV protease, an enzyme essential for viral replication. To date, more than 2,500 people with genotype 1 hepatitis C have received telaprevir in Phase 2 and Phase 3 studies.

Vertex is developing telaprevir in collaboration with Tibotec BVBA and Mitsubishi Tanabe Pharma. Vertex has rights to commercialize telaprevir in North America. through its affiliate, Janssen, Tibotec has rights to commercialize telaprevir in Europe, South America, Australia, the Middle East and certain other countries. Mitsubishi Tanabe Pharma has rights to commercialize telaprevir in Japan and certain far East countries.

Hepatitis C is a serious liver disease caused by the hepatitis C virus, which is spread through direct contact with the blood of infected people and ultimately affects the liver.1 Chronic hepatitis C can lead to serious and life-threatening liver problems, including liver damage, cirrhosis, liver failure or liver cancer.1 Though many people with hepatitis C may not experience symptoms, others may have symptoms such as fatigue, fever, jaundice and abdominal pain.1 Approximately 60 percent of genotype 1 patients who undergo an initial 48-week regimen with pegylated-interferon and ribavirin, the currently approved medicines, do not achieve SVR,2,3,4 or viral cure.5 if treatment is not successful and a person does not achieve a viral cure, they remain at an increased risk for progressive liver disease.6,7,8.9,10

Hepatitis C in the United States

Up to 3.9 million people in the United States have chronic hepatitis C and 75 percent of them are unaware of their infection.11 the majority of people with hepatitis C in the U.S. were born between 1946 and 1964, accounting for two of every three people with chronic hepatitis C.10 Hepatitis C is the leading cause of liver transplantations in the U.S. and is reported to contribute to 4,600 to 12,000 deaths annually.7 by 2029, total annual medical costs in the U.S. for people with hepatitis C are expected to more than double, from $30 billion in 2009 to approximately $85 billion.10

Hepatitis C in Canada

Approximately 250,000 people in Canada have chronic hepatitis C and more than a third of them do not know they are infected.12 three provinces account for 80 percent of hepatitis C infections in Canada: Ontario (42 percent), British Columbia (22 percent) and Quebec (16 percent).13 each year up to 5,000 people are newly infected with hepatitis C and in 2007 alone, nearly 8,000 people were infected.12, 13 in 2010, the annual cost of hepatitis C due to medical treatment and lost productivity in Canada was estimated to reach $1 billion.14 by 2022, the number of hepatitis C-related deaths is expected to increase by one-third.15

Additional resources for media are available at: investors.vrtx.com/press.cfm.

PEGASYS® and COPEGUS® are registered trademarks of Hoffman-LA Roche.

Special Note regarding Forward-looking Statements

This press release contains forward-looking statements, including statements regarding (i) the FDA’s target review date for the telaprevir NDA, (ii) Priority Review in Canada allowing for faster review of new Drug Submissions, typically shortening the review time to approximately six to nine months and (iii) Vertex working with the FDA and Health Canada to make telaprevir available as quickly as possible for people with hepatitis C. while the company believes the forward-looking statements contained in this press release are accurate, there are a number of factors that could cause actual events or results to differ materially from those indicated by such forward-looking statements. Those risks and uncertainties include, among other things, that Vertex could experience unforeseen delays in obtaining approval to market telaprevir; that there may be varying interpretations of the data from the telaprevir clinical trials; that future outcomes from clinical trials of telaprevir may not be favorable; that future scientific, clinical, competitive or other market factors may adversely affect the potential for telaprevir-based therapy and the other risks listed under Risk Factors in Vertex’s annual report and quarterly reports filed with the Securities and Exchange Commission and available through Vertex’s website at vrtx.com. Vertex disclaims any obligation to update the information contained in this press release as new information becomes available.

Vertex creates new possibilities in medicine. Our team aims to discover, develop and commercialize innovative therapies so people with serious diseases can lead better lives.

Vertex scientists and our collaborators are working on new medicines to cure or significantly advance the treatment of hepatitis C, cystic fibrosis, epilepsy and other life-threatening diseases.

Founded more than 20 years ago in Cambridge, MA, we now have ongoing worldwide research programs and sites in the U.S., U.K. and Canada.

About Vertex in Canada

In 2009, Vertex established a research and development site in Laval, Quebec through the acquisition of Virochem Pharma, Inc. Vertex is expanding its existing research and development infrastructure with the addition of commercial and medical teams to support the potential launch of telaprevir in Canada.

For more information and to view Vertex’s press releases, please visit vrtx.com.

Most people have no symptoms when they are first infected with the hepatitis C virus. if you do develop symptoms, they may include:

Feeling very tired.
Joint pain.
Belly pain.
Itchy skin.
Sore muscles.
Dark urine.
Yellowish eyes and skin (jaundice). Jaundice usually appears only after other symptoms have started to go away.
Most people go on to develop chronic hepatitis C but still do not have symptoms. this makes it common for people to have hepatitis C for 15 years or longer before it is diagnosed.