By Lisa Richwine
ADELPHI, Md., Nov 16 (Reuters) - U.S. advisers are considering whether to recommend approval of a highly anticipated lupus treatment from Human Genome Sciences Inc amid concerns the drug might increase suicides or carry other serious risks.
Benlysta could be the first medicine in a half-century to win approval for fighting the debilitating and potentially fatal autoimmune disease. Annual global sales are forecast at $2.2 billion by 2014, according to Thomson Reuters.
That would transform unprofitable biotech Human Genome into an industry star.
The company would split Benlysta profits with partner GlaxoSmithKline Plc.
A Food and Drug Administration advisory panel, set to vote later on Tuesday, heard pitches from Human Genome and patients who pushed for the medicine's approval. But FDA reviewers questioned how much benefit the drug offered and noted potential risks.
Human Genome argued the intravenous drug was an important advance for fighting lupus, a disease that causes the immune system to attack the body's own tissue and organs. That can lead to arthritis, kidney damage, chest pain, fatigue, skin rash and other problems. Organ damage can be fatal, and current treatments have severe side effects.
Lupus patients have 'persevered through a decade of failed trials,' said Dr. Michelle Petri, a rheumatologist at Johns Hopkins University and consultant to Human Genome. Benlysta offers 'compelling efficacy' with a 'favorable safety profile,' she told the panel.
Forty-six-year-old Donna Flenory said lupus caused severe fatigue and joint pain that forced her to give up her career as an occupational therapist. High doses of steroids improved her symptoms for a time but repeated flare-ups sent her to the hospital every year, usually in the autumn.
After joining a study of Benlysta, 'when fall rolled around I was no longer sick. I had a life again,' she told the panel, adding 'please realize we as patients need something.'
FDA reviewers, however, suggested Benlysta might have only a modest effect on lupus symptoms and might raise the risk of death, infection, or psychiatric problems including suicide. Two patients treated with Benlysta during clinical trials killed themselves and there were no suicides with a placebo.
ANALYSTS PREDICT APPROVAL
Industry analysts said ahead of the meeting that they expected the advisory panel to endorse the drug and the FDA to ultimately approve it for sale. Many said lupus patients already faced serious health problems, and there was no clear evidence Benlysta contributed to deaths or suicides.
Investors sent Human Genome shares down 11 percent on Friday after the FDA released preliminary staff comments. The stock rebounded on Monday, closing up 9.7 percent at $25.88 on Nasdaq, and was halted on Tuesday while the FDA panel met.
The company's shares remain far above the March 2009 level of less than 50 cents, seen before the first encouraging Benlysta results were released.
(For a graphic on how prospects for Benlysta have affected Human Genome shares, click on http://r.reuters.com/sed35q)
The FDA panel is set to vote on three questions: whether Benlysta is effective, safe and should be approved.
The panel's input is key because the FDA usually follows those recommendations when deciding whether to approve new medicines. A final FDA ruling is due by Dec. 9, although some analysts forecast a decision could be delayed by three months.
Patients, many speaking through tears, pleaded with the panel to back Benlysta. An estimated 5 million people worldwide have lupus, and many cannot find relief with current drugs or suffer harsh side effects such as severe bone loss from steroids.
Benlysta, known generically as belimumab, is given once a month by intravenous infusion. Analysts expect the drug would cost a patient between $15,000 and $30,000 annually, a price tag in line with biotech medicines for other autoimmune diseases.
The drug is designed to inhibit BLyS, a protein in the body that exists to keep B-cells functioning normally. B-cells make antibodies that prevent infection. In lupus patients, B-cells are overstimulated and make antibodies that attack the body.
(Editing by Maureen Bavdek, Lisa Von Ahn and Tim Dobbyn) Keywords: HUMANGENOME LUPUS/ (lisa.richwine@thomsonreuters.com +1 202 310 5691; Reuters Messaging: lisa.richwine.reuters.com@reuters.net) COPYRIGHT Copyright Thomson Reuters 2010. All rights reserved. The copying, republication or redistribution of Reuters News Content, including by framing or similar means, is expressly prohibited without the prior written consent of Thomson Reuters.
ADELPHI, Md., Nov 16 (Reuters) - U.S. advisers are considering whether to recommend approval of a highly anticipated lupus treatment from Human Genome Sciences Inc amid concerns the drug might increase suicides or carry other serious risks.
Benlysta could be the first medicine in a half-century to win approval for fighting the debilitating and potentially fatal autoimmune disease. Annual global sales are forecast at $2.2 billion by 2014, according to Thomson Reuters.
That would transform unprofitable biotech Human Genome into an industry star.
The company would split Benlysta profits with partner GlaxoSmithKline Plc.
A Food and Drug Administration advisory panel, set to vote later on Tuesday, heard pitches from Human Genome and patients who pushed for the medicine's approval. But FDA reviewers questioned how much benefit the drug offered and noted potential risks.
Human Genome argued the intravenous drug was an important advance for fighting lupus, a disease that causes the immune system to attack the body's own tissue and organs. That can lead to arthritis, kidney damage, chest pain, fatigue, skin rash and other problems. Organ damage can be fatal, and current treatments have severe side effects.
Lupus patients have 'persevered through a decade of failed trials,' said Dr. Michelle Petri, a rheumatologist at Johns Hopkins University and consultant to Human Genome. Benlysta offers 'compelling efficacy' with a 'favorable safety profile,' she told the panel.
Forty-six-year-old Donna Flenory said lupus caused severe fatigue and joint pain that forced her to give up her career as an occupational therapist. High doses of steroids improved her symptoms for a time but repeated flare-ups sent her to the hospital every year, usually in the autumn.
After joining a study of Benlysta, 'when fall rolled around I was no longer sick. I had a life again,' she told the panel, adding 'please realize we as patients need something.'
FDA reviewers, however, suggested Benlysta might have only a modest effect on lupus symptoms and might raise the risk of death, infection, or psychiatric problems including suicide. Two patients treated with Benlysta during clinical trials killed themselves and there were no suicides with a placebo.
ANALYSTS PREDICT APPROVAL
Industry analysts said ahead of the meeting that they expected the advisory panel to endorse the drug and the FDA to ultimately approve it for sale. Many said lupus patients already faced serious health problems, and there was no clear evidence Benlysta contributed to deaths or suicides.
Investors sent Human Genome shares down 11 percent on Friday after the FDA released preliminary staff comments. The stock rebounded on Monday, closing up 9.7 percent at $25.88 on Nasdaq, and was halted on Tuesday while the FDA panel met.
The company's shares remain far above the March 2009 level of less than 50 cents, seen before the first encouraging Benlysta results were released.
(For a graphic on how prospects for Benlysta have affected Human Genome shares, click on http://r.reuters.com/sed35q)
The FDA panel is set to vote on three questions: whether Benlysta is effective, safe and should be approved.
The panel's input is key because the FDA usually follows those recommendations when deciding whether to approve new medicines. A final FDA ruling is due by Dec. 9, although some analysts forecast a decision could be delayed by three months.
Patients, many speaking through tears, pleaded with the panel to back Benlysta. An estimated 5 million people worldwide have lupus, and many cannot find relief with current drugs or suffer harsh side effects such as severe bone loss from steroids.
Benlysta, known generically as belimumab, is given once a month by intravenous infusion. Analysts expect the drug would cost a patient between $15,000 and $30,000 annually, a price tag in line with biotech medicines for other autoimmune diseases.
The drug is designed to inhibit BLyS, a protein in the body that exists to keep B-cells functioning normally. B-cells make antibodies that prevent infection. In lupus patients, B-cells are overstimulated and make antibodies that attack the body.
(Editing by Maureen Bavdek, Lisa Von Ahn and Tim Dobbyn) Keywords: HUMANGENOME LUPUS/ (lisa.richwine@thomsonreuters.com +1 202 310 5691; Reuters Messaging: lisa.richwine.reuters.com@reuters.net) COPYRIGHT Copyright Thomson Reuters 2010. All rights reserved. The copying, republication or redistribution of Reuters News Content, including by framing or similar means, is expressly prohibited without the prior written consent of Thomson Reuters.