Month: June 2021

New drug for severe form of MS generates glimmer of hope — and tempered expectations

first_imgPoliticsNew drug for severe form of MS generates glimmer of hope — and tempered expectations MRI brain scan from a patient with multiple sclerosis. Dr. W. Crum/Tim Beddow/Science Source Related: There is finally some progress in the hunt for a treatment for the most difficult form of multiple sclerosis — the form that has stubbornly resisted every attempt to find a therapy.The catch, experts say, is that people with this form of the disease should keep their expectations low, because even the new drug that’s now in the last stages of development doesn’t appear to have a big clinical impact.The reason that the new drug is generating so much interest, though, is that it’s the first time a therapy for primary progressive MS has had any impact at all.advertisement ‘Fearful for the future’: Father, daughter fight different forms of MS The FDA has approved 13 drugs for treating relapsing-remitting MS, but none have been shown to work for people with primary progressive disease. The new “breakthrough” drug, called ocrelizumab, is one of six that are in various stages of development for this recalcitrant form, one that affects 10 to 15 percent of the estimated 400,000 Americans living with MS.On Friday, the company behind ocrelizumab, the Roche subsidiary Genentech, will release new Phase 3 trial data at a conference of the Americas Committee for Treatment and Research in Multiple Sclerosis, which is being held in New Orleans.Genentech has already published a first round of the results, which showed some impact on a wide range of measures for people with primary progressive MS. The new data will include more detail on how ocrelizumab affected subgroups of patients, as well as new information on how it worked in people with relapsing-remitting MS, to show whether it can also work on the more common variety of the disease.These new results will demonstrate that the drug “appears to have efficacy across the patient population” of people with primary progressive MS, including people who had active inflammation and those who didn’t, according to Dr. Peter Chin, principal medical director for neuroscience at Genentech. It will also show a “significant reduction” in the major markers of the disease for people with relapsing-remitting MS, he said.Even before those data are released, the fact that the FDA has given the coveted “breakthrough” designation to the drug has given new hope to people who have been waiting for a treatment for years.“When I found out about this this morning, it was like every cell in my body smiled,” said Gigi Ranno, an outreach coordinator for the Massachusetts Department of Conservation and Recreation, who has primary progressive MS.“I’ve had MS for over 25 years. It’s the first time that I have heard that there’s a drug that’s this close to being submitted to the FDA for approval for my kind of MS,” she said in an interview Thursday.Gigi Ranno uses a wheelchair, even for sailing, owing to her primary progressive MS. John Tlumacki/The Boston GlobeThe FDA announcement has also generated excitement among advocates like Tim Coetzee, the chief advocacy, services, and research officer at the National Multiple Sclerosis Society. “We’ve had so many failures with treatments for people with primary progressive MS that this really represents a source of hope,” he said.Experts do warn, however, that even if the drug is approved after the fast-track review, people with primary progressive MS shouldn’t expect spectacular results.“It is truly a landmark, and really provides hope to people who are living with primary progressive MS. That being said, the results are on the modest side,” said Dr. Tanuja Chitnis, a neurologist at the Ann Romney Center for Neurologic Diseases at Brigham and Women’s Hospital in Boston, who was not involved in the drug trials. And it’s the first MS drug to ever receive a “breakthrough” designation from the Food and Drug Administration, which puts the experimental medicine on a fast track to get through the review process as quickly as possible. The agency granted the designation earlier this week. By David Nather Feb. 19, 2016 Reprints Ocrelizumab works by targeting a certain kind of immune cell that can contribute to the damage to the nervous system, including the nerve cells and the protective covering around nerve fibers. It is taken by intravenous infusion twice a year.In the clinical trial data released last fall, which covered 732 people with primary progressive MS, people who took the drug had a 24 percent reduction in the progression of disability after 12 weeks compared to those who received a placebo. Study participants on the experimental medicine also experienced slower declines in walking ability and brain volume than people in the control group.These differences, while undramatic, were statistically significant, which is the main threshold the FDA will be looking for when it decides whether to approve the medication. “It’s a positive result when you’ve had 20 years of failure,” said Coetzee.Based on the study results, Dr. Dennis Bourdette, executive director of the Oregon Health and Science University’s Multiple Sclerosis Center, said it was “very appropriate” for the FDA to grant breakthrough status to the drug and that he was “hopeful that it will be approved for the treatment of primary progressive MS.”But Bourdette, who was not involved in the Genentech-funded trials, also noted that the patients in the study were younger than average for people with primary progressive MS, which usually begins about 10 years later than relapsing-remitting MS. Younger patients could be more responsive to treatment, and “it will be uncertain how effective ocrelizumab will be for older and more disabled patients,” he said.For Michael Bunting, 66, it’s more of a sign of distant hope than something that could give him immediate relief.Bunting, who talked to STAT last year about his battles with progressive MS, has a different variation of the disease: secondary progressive MS. That’s a kind that often begins with relapsing-remitting MS and then turns into a steady deterioration. So even if the new drug is approved, it’s not something that would necessarily help him because it’s not yet been evaluated for people with his form of the disease.Still, Bunting sees it as a big step for all MS patients, and one that could lead to more research that could eventually find a drug that would help him.“If this thing helps just in the primary progressive, that’s great, because it will help us in the long run [to] finally find something that will help the secondary,” Bunting said.“I just hope they don’t wait too long.” A progress report: Fighting the deadliest diseases, one step at a time Related: Tags FDAmultiple sclerosisneurologypharmaceuticalspolicy Primary progressive MS is a relatively rare form of the neurological disease in which a person doesn’t have severe attacks, the way people do with the more common, relapsing-remitting kind. Instead, they suffer a slow, steady physical deterioration with no hope of recovery.advertisementlast_img read more

Drugs to combat hair loss could raise risk for erectile dysfunction, study finds

first_imgThen he ran all sorts of statistical tests, and found that, among men who did not have prostate disease, those taking either drug for more than 208.5 days, or about seven months, had a statistically significant higher chance of developing “persistent erectile dysfunction” (which means the problem lasts for more than 90 days after you stop taking the drug).It’s important to note that the rate of persistent erectile dysfunction in both groups was still quite low. Just 1.3 percent of men who took one of the drugs for more than 208.5 days had that problem. As did just 0.44 percent of the group that took one of the drugs for less time.advertisement What is actually in erectile dysfunction supplements? Why I’d never take the hair loss drug President Trump uses By Ike Swetlitz March 9, 2017 Reprints Related: Quick TakeDrugs to combat hair loss could raise risk for erectile dysfunction, study finds The presidential prescription:Side note: One of the most famous patients on this type of drug may be President Trump. In February, the New York Times reported that the president takes a “small dose” of finasteride, citing Dr. Harold Bornstein, who served for decades as his personal doctor.The bottom line:Drugs designed to treat baldness and an enlarged prostate may have long-lasting effects on men’s reproductive health, but more research will be needed.center_img Men who take a popular class of drugs to combat hair loss or prostate shrinkage may be at a small increased risk for long-lasting erectile dysfunction if they take the drug for a long time, a new study found.Why it matters:The FDA label for Propecia, one of the drugs in question, does note that adverse reactions include “sexual dysfunction that continued after discontinuation of treatment, including erectile dysfunction, libido disorders, ejaculation disorders, and orgasm disorders.” Still, Dr. Steven Belknap, a research assistant professor at the Northwestern University Feinberg School of Medicine, said men don’t know enough about possible side effects. He’s the senior author on the paper published Thursday in PeerJ.The nitty-gritty:Motivated by a buddy who asked for his professional advice about whether he should take the drug finasteride for baldness, Belknap scoured patient records at Northwestern. He looked for all men who were taking either finasteride or dutasteride, which work in similar ways in the body, and who did not have erectile dysfunction. He found 11,909 of them.advertisement But keep in mind:The study relied on patient records, so it has weaknesses.Some men may have stopped taking finasteride immediately after experiencing negative reactions, which could have the effect of minimizing Belknap’s count of unpleasant side effects. On the other hand, his count could be inflated if he erroneously included in his study men who had erectile dysfunction even before they started taking the drugs, but didn’t have that information recorded in their medical records.The study was funded in part by the Post-Finasteride Syndrome Foundation, a nonprofit that funds research to help men who have taken finasteride and experienced “persistent sexual, neurological, and physical adverse reactions,” according to the organization’s website. Additional funding came from the National Institutes of Health.What they’re saying:Researchers unaffiliated with the study could not be reached for comment Wednesday evening.But others have published related research with similar findings. A 2015 paper, for instance, found that men with enlarged prostates who took finasteride experienced a decrease in erectile function as well as a decrease in testosterone levels over four years. APStock Related: Tags men’s healthsexual healthlast_img read more

White House panel urges Trump to declare state of emergency over opioid crisis

first_img What’s included? @levfacher [email protected] What is it? Politics GET STARTED Log In | Learn More WASHINGTON — The White House’s commission on combating the opioid epidemic has recommended that President Trump declare a federal state of emergency to address the crisis, a potentially significant step for an administration that has repeatedly pledged to take steps to ease the epidemic.“The first and most urgent recommendation of this Commission is direct and completely within your control. Declare a national emergency under either the Public Health Service Act or the Stafford Act,” the committee wrote in an interim report released Monday. Tags addictionopioidspolicy Washington Correspondent Lev Facher covers the politics of health and life sciences.center_img Unlock this article by subscribing to STAT+ and enjoy your first 30 days free! GET STARTED White House panel urges Trump to declare state of emergency over opioid crisis Daily reporting and analysis The most comprehensive industry coverage from a powerhouse team of reporters Subscriber-only newsletters Daily newsletters to brief you on the most important industry news of the day STAT+ Conversations Weekly opportunities to engage with our reporters and leading industry experts in live video conversations Exclusive industry events Premium access to subscriber-only networking events around the country The best reporters in the industry The most trusted and well-connected newsroom in the health care industry And much more Exclusive interviews with industry leaders, profiles, and premium tools, like our CRISPR Trackr. Lev Facher By Lev Facher July 31, 2017 Reprints STAT+ is STAT’s premium subscription service for in-depth biotech, pharma, policy, and life science coverage and analysis. Our award-winning team covers news on Wall Street, policy developments in Washington, early science breakthroughs and clinical trial results, and health care disruption in Silicon Valley and beyond. About the Author Reprints Spencer Platt/Getty Imageslast_img read more

Amicus therapy shows durable muscle function improvements in Pompe disease patients

first_img About the Author Reprints John Crowley and his daughter, Megan, whose illness inspired him to look for a treatment. Crowley family @adamfeuerstein GET STARTED Amicus Therapeutics (FOLD) is presenting updated results Wednesday from a small clinical trial involving patients with the rare and debilitating Pompe disease. Amicus’ drug continues to improve walking performance and stabilize lung function in patients followed for up to one year.Data from this same Pompe disease study were last presented by Amicus in October. Daily reporting and analysis The most comprehensive industry coverage from a powerhouse team of reporters Subscriber-only newsletters Daily newsletters to brief you on the most important industry news of the day STAT+ Conversations Weekly opportunities to engage with our reporters and leading industry experts in live video conversations Exclusive industry events Premium access to subscriber-only networking events around the country The best reporters in the industry The most trusted and well-connected newsroom in the health care industry And much more Exclusive interviews with industry leaders, profiles, and premium tools, like our CRISPR Trackr. Log In | Learn More What is it? STAT+ is STAT’s premium subscription service for in-depth biotech, pharma, policy, and life science coverage and analysis. Our award-winning team covers news on Wall Street, policy developments in Washington, early science breakthroughs and clinical trial results, and health care disruption in Silicon Valley and beyond. What’s included?center_img By Adam Feuerstein Feb. 7, 2018 Reprints [email protected] Adam Feuerstein Senior Writer, Biotech Adam is STAT’s national biotech columnist, reporting on the intersection of biotech and Wall Street. He’s also a co-host of “The Readout LOUD” podcast. Unlock this article — plus daily coverage and analysis of the biotech sector — by subscribing to STAT+. First 30 days free. GET STARTED Amicus therapy shows durable muscle function improvements in Pompe disease patients Biotech Tags biotechdrug developmentrare diseaseSTAT+last_img read more

McCaskill seeks to end tax breaks for consumer drug ads

first_img McCaskill seeks to end tax breaks for consumer drug ads Pharmalot What’s included? Ed Silverman [email protected] Once again, a congressional lawmaker is introducing legislation that would end the tax break that drug makers can take for advertising medicines to consumers.Called the End Taxpayer Subsidies for Drug Ads Act, the bill reflects concerns that drug makers are spending too much to market high-priced medicines at a time of growing anger over the cost of prescription drugs, according to U.S. Sen. Claire McCaskill (D- Mo.), who introduced the bill. She noted that only the U.S. and New Zealand allow consumer advertising of pharmaceuticals. GET STARTED Pharmalot Columnist, Senior Writer Ed covers the pharmaceutical industry. About the Author Reprints By Ed Silverman March 1, 2018 Reprintscenter_img Daily reporting and analysis The most comprehensive industry coverage from a powerhouse team of reporters Subscriber-only newsletters Daily newsletters to brief you on the most important industry news of the day STAT+ Conversations Weekly opportunities to engage with our reporters and leading industry experts in live video conversations Exclusive industry events Premium access to subscriber-only networking events around the country The best reporters in the industry The most trusted and well-connected newsroom in the health care industry And much more Exclusive interviews with industry leaders, profiles, and premium tools, like our CRISPR Trackr. Log In | Learn More Unlock this article — plus daily coverage and analysis of the pharma industry — by subscribing to STAT+. First 30 days free. GET STARTED Tags pharmaceuticalsSTAT+ STAT+ is STAT’s premium subscription service for in-depth biotech, pharma, policy, and life science coverage and analysis. Our award-winning team covers news on Wall Street, policy developments in Washington, early science breakthroughs and clinical trial results, and health care disruption in Silicon Valley and beyond. Dom Smith/STAT @Pharmalot What is it?last_img read more

In rare advisory, surgeon general urges public to carry overdose-reversal medication

first_img General Assignment Reporter Andrew covers a range of topics, from addiction to public health to genetics. In rare advisory, surgeon general urges public to carry overdose-reversal medication [email protected] Surgeon General Jerome Adams is issuing a rare public health advisory on Thursday, calling for friends and family of people at risk for opioid overdoses to carry the OD-reversal medication naloxone. He likened the treatment to other livesaving interventions, such as knowing how to perform CPR or use an EpiPen.The recommendation comes in the form of a surgeon general’s advisory, a tool used to draw attention to major public health issues. The last one, focused on drinking during pregnancy, was issued in 2005. GET STARTED Unlock this article — and get additional analysis of the technologies disrupting health care — by subscribing to STAT+. First 30 days free. GET STARTED What’s included? STAT+ is STAT’s premium subscription service for in-depth biotech, pharma, policy, and life science coverage and analysis. Our award-winning team covers news on Wall Street, policy developments in Washington, early science breakthroughs and clinical trial results, and health care disruption in Silicon Valley and beyond. Daily reporting and analysis The most comprehensive industry coverage from a powerhouse team of reporters Subscriber-only newsletters Daily newsletters to brief you on the most important industry news of the day STAT+ Conversations Weekly opportunities to engage with our reporters and leading industry experts in live video conversations Exclusive industry events Premium access to subscriber-only networking events around the country The best reporters in the industry The most trusted and well-connected newsroom in the health care industry And much more Exclusive interviews with industry leaders, profiles, and premium tools, like our CRISPR Trackr. By Andrew Joseph April 5, 2018 Reprintscenter_img Andrew Joseph Tags addictionopioidspolicy About the Author Reprints What is it? Health Surgeon General Jerome Adams Chip Somodevilla/Getty Images @DrewQJoseph Log In | Learn More last_img read more

Celgene’s beaten-down stock is cheap, but only if investors regain trust in its financial outlook

first_img @adamfeuerstein By Adam Feuerstein April 30, 2018 Reprints Log In | Learn More Tags biotechfinanceSTAT+ Daily reporting and analysis The most comprehensive industry coverage from a powerhouse team of reporters Subscriber-only newsletters Daily newsletters to brief you on the most important industry news of the day STAT+ Conversations Weekly opportunities to engage with our reporters and leading industry experts in live video conversations Exclusive industry events Premium access to subscriber-only networking events around the country The best reporters in the industry The most trusted and well-connected newsroom in the health care industry And much more Exclusive interviews with industry leaders, profiles, and premium tools, like our CRISPR Trackr. About the Author Reprints Celgene’s beaten-down stock is cheap, but only if investors regain trust in its financial outlook GET STARTED STAT+ is STAT’s premium subscription service for in-depth biotech, pharma, policy, and life science coverage and analysis. Our award-winning team covers news on Wall Street, policy developments in Washington, early science breakthroughs and clinical trial results, and health care disruption in Silicon Valley and beyond. What’s included?center_img Unlock this article by subscribing to STAT+ and enjoy your first 30 days free! GET STARTED Adam Feuerstein What is it? Kristoffer Tripplaar/SIPA via AP Senior Writer, Biotech Adam is STAT’s national biotech columnist, reporting on the intersection of biotech and Wall Street. He’s also a co-host of “The Readout LOUD” podcast. [email protected] I hesitate to call any single earnings report a do-or-die event, but for troubled Celgene (CELG), Friday’s first-quarter financial update comes pretty close.Celgene CEO Mark Alles and his team must convince investors that the horrible run of mistakes that started last fall is finally over and won’t happen again. A positive regulatory update on the delayed multiple sclerosis drug candidate ozanimod would be welcome news. Adam’s Take last_img read more

Watchdog asks ethics officials to probe Azar over industry ties and views on rebates

first_img Log In | Learn More Watchdog asks ethics officials to probe Azar over industry ties and views on rebates STAT+ is STAT’s premium subscription service for in-depth biotech, pharma, policy, and life science coverage and analysis. Our award-winning team covers news on Wall Street, policy developments in Washington, early science breakthroughs and clinical trial results, and health care disruption in Silicon Valley and beyond. Pharmalot Columnist, Senior Writer Ed covers the pharmaceutical industry. A watchdog group has asked ethics officials at the Department of Health and Human Services to investigate the relationship between Alex Azar, who heads the department, and Eli Lilly (LLY), his former employer, over a recently proposed rule that would benefit drug makers.The request by the Campaign for Accountability, a nonprofit, comes in response to a proposal the Trump administration is considering to reduce or restrict rebates, which are essentially a type of discount that drug makers provide pharmacy benefit managers off the wholesale, or list, price for their medicines in order to receive favorable placement on formularies, which are lists of insured drugs. About the Author Reprints By Ed Silverman Aug. 28, 2018 Reprints What is it? [email protected] What’s included?center_img Pharmalot Ed Silverman Tags drug pricingpharmaceuticalspolicySTAT+ Unlock this article by subscribing to STAT+ and enjoy your first 30 days free! GET STARTED GET STARTED Daily reporting and analysis The most comprehensive industry coverage from a powerhouse team of reporters Subscriber-only newsletters Daily newsletters to brief you on the most important industry news of the day STAT+ Conversations Weekly opportunities to engage with our reporters and leading industry experts in live video conversations Exclusive industry events Premium access to subscriber-only networking events around the country The best reporters in the industry The most trusted and well-connected newsroom in the health care industry And much more Exclusive interviews with industry leaders, profiles, and premium tools, like our CRISPR Trackr. Tasos Katopodis/Getty Images @Pharmalot last_img read more

Watch: The state of biotech at time of innovation — and high prices

first_img CAMBRIDGE, Mass. — We live in an age of miracles, but they come at a high cost, with new gene therapies costing millions of dollars each. Can we afford them? STAT senior writer Adam Feuerstein sat down with three industry CEOs to discuss this and other questions during a session called ‘The State of Biotech” at the inaugural STAT summit in Cambridge this week.Dr. Athena Countouriotis, the president and chief executive officer of Turning Point Therapeutics, spoke about the price Roche set for a lung cancer drug, Rozlytrek. The drug currently costs about $17,050 per month; an earlier drug cost $15,000, while one competing drug, Vitrakvi, costs about twice as much.“Many people thought, well, if it’s really innovative and very different, they could definitely [put a] premium price on that. So I give Roche a little bit of credit,” she said.advertisement Tags biotechnologySTAT+ About the Author Reprints STAT SummitWatch: The state of biotech at time of innovation — and high prices Jeff Jonas, CEO of Sage Therapeutics, was also emphatic that strict drug pricing policies could make the broader biopharma industry will suffer — and that drafting regulations based on “bad actors who we haven’t called out” could be an overreaction.Watch the full discussion below.advertisement STAT Summit: The State Of BiotechVolume 90%Press shift question mark to access a list of keyboard shortcutsKeyboard ShortcutsEnabledDisabledPlay/PauseSPACEIncrease Volume↑Decrease Volume↓Seek Forward→Seek Backward←Captions On/OffcFullscreen/Exit FullscreenfMute/UnmutemSeek %0-9 facebook twitter Email Linkhttps://www.statnews.com/2019/11/22/watch-the-state-of-biotech-at-time-of-innovation-and-high-prices/?jwsource=clCopied EmbedCopiedLive00:0032:0332:03  New gene therapies cost millions of dollars each. Can we afford them? STAT reporter Adam Feuerstein discusses the state of biotech industry with Dr. Athena Countouriotis of Turning Point Therapeutics, Dr. Jeff Jonas of Sage Therapeutics, and Nick Leschly of Bluebird. STATcenter_img [email protected] STAT staff From left, Nick Leschly, CEO of Bluebird Bio; Jeff Jonas, CEO of Sage Therapeutics; and Athena M. Countouriotis, CEO of Turning Point Therapeutics. Emilie Pickering for STAT By STAT staff Nov. 22, 2019 Reprintslast_img read more

As Trump looks overseas for drug pricing ideas, one expert says France has some useful approaches

first_img GET STARTED As the U.S. grapples with rising costs for prescription medicines, the Trump administration has floated a proposal that would have Medicare use a so-called International Pricing Index as a benchmark to pay for certain drugs. Although still being crafted, the idea has, once again, focused attention on the different prices paid in the U.S. and other countries. So Suffolk University professor Marc Rodwin, who specializes in health law, has begun studying payment systems elsewhere and recently looked at France, where retail drug spending declined between 2008 and 2017, compared with rising spending in the U.S. We spoke with him about the different approaches taken by the two countries and what lessons can be learned. This is an edited version of our conversation.Pharmalot: What prompted this and why start with France? A pharmacy in Paris. STEPHANE DE SAKUTIN/AFP/Getty Images What is it? Pharmalot [email protected] Unlock this article — plus daily coverage and analysis of the pharma industry — by subscribing to STAT+. First 30 days free. GET STARTED As Trump looks overseas for drug pricing ideas, one expert says France has some useful approaches Ed Silverman STAT+ is STAT’s premium subscription service for in-depth biotech, pharma, policy, and life science coverage and analysis. Our award-winning team covers news on Wall Street, policy developments in Washington, early science breakthroughs and clinical trial results, and health care disruption in Silicon Valley and beyond.center_img Daily reporting and analysis The most comprehensive industry coverage from a powerhouse team of reporters Subscriber-only newsletters Daily newsletters to brief you on the most important industry news of the day STAT+ Conversations Weekly opportunities to engage with our reporters and leading industry experts in live video conversations Exclusive industry events Premium access to subscriber-only networking events around the country The best reporters in the industry The most trusted and well-connected newsroom in the health care industry And much more Exclusive interviews with industry leaders, profiles, and premium tools, like our CRISPR Trackr. What’s included? About the Author Reprints Tags BostonDonald Trumpdrug developmentdrug pricingMedicareSTAT+ Pharmalot Columnist, Senior Writer Ed covers the pharmaceutical industry. Log In | Learn More @Pharmalot By Ed Silverman Dec. 3, 2019 Reprintslast_img read more