Neurocrine Biosciences, Inc. (NASDAQ: NBIX) today announced that the U.S. Food and Drug Administration (FDA) has accepted its New Drug Application (NDA) for opicapone, a novel, once-daily, oral, selective catechol-O-methyltransferase (COMT) inhibitor as an adjunctive treatment to levodopa/carbidopa in patients with Parkinson’s disease experiencing OFF episodes. Parkinson’s disease is a chronic, progressive and debilitating neurodegenerative disorder that affects approximately one million people in the U.S. The FDA has set a standard 12-month review process with a Prescription Drug User Fee Act (PDUFA) target action date of April 26, 2020.
Wednesday, July 10, 2019
Fresenius: U.S. plans for changes to kidney disease care positive for strategy
Fresenius Medical Care, the world’s largest provider of dialysis products and services, is pleased the U.S. administration’s plans for changing the way care is provided to people with kidney disease supports its existing strategy. The company has long worked on various initiatives to promote home dialysis, improve access to transplants, and develop new, value-based care models for chronic kidney disease patients.
Rice Powell, CEO of Fresenius Medical Care, said: ‘We congratulate the Administration on today’s announcement and celebrate the proposed initiatives as a win for our patients and for the 30 million Americans living with kidney disease. We are committed to continuously improving the quality of life of patients affected by kidney disease and have already established initiatives to improve prevention, offer more flexible treatment options, introduce value-based care models and promote organ donation -in the United States and abroad.
We share the U.S. Administration’s commitment to expanding access to home dialysis, transplantation and new models of value-based care for chronic kidney disease, and we see it as an endorsement of our initiatives. We invest constantly in innovation and will continue to do so in order to further develop the healthcare system. The proposed reimbursement models and new incentives will help foster further innovation and support a healthcare delivery system structure that is closely attuned to the needs of our patients.
Our recent merger with NxStage, which makes the leading hemodialysis machine for home use, is just one piece of a focused effort to educate patients and physicians around the benefits of home treatment and provide industry leading solutions to enable them to do so. We are also investing in technologies for the future, including new innovations for remote patient monitoring and telehealth that, combined with predictive analytics and artificial intelligence, will make it easier to help patients between visits to a doctor and avoid unnecessary hospitalizations.
We welcome reimbursement reforms that facilitate investments in care models designed to improve outcomes and help reduce costs, two goals to which more use of home dialysis and transplants can equally contribute. We will carefully review the U.S. administration’s proposal and contribute to developing the framework that offers the best possible conditions and greatest benefit for patients.’
DaVita Statement on Advancing American Kidney Health
DaVita Kidney Care appreciates President Trump, the Administration and Secretary Azar for taking steps to improve the lives of kidney patients. We appreciate the Administration’s willingness to collaborate with the industry to ensure these new models address patient choice, prevention and transplantation.
Statement Attributable to Javier Rodriguez, CEO for DaVita Inc.:‘DaVita is encouraged that this Administration has taken steps toward holistic, value-based care for kidney patients. We have pushed for progressive policies to give all patients access to integrated kidney care, the benefits of which are significant to our patient population. Educating patients about kidney disease is critical to prevention and slowing its progression. Early intervention leads patients who may still need dialysis to choose the best treatment option for their lifestyle and reduces expensive hospitalizations.
In partnership with nephrologists, we are best positioned to deliver in the home dialysis space, as the largest provider of home dialysis in the U.S. We’re accelerating home growth with our investments in technologies, such as home remote monitoring and a telehealth platform, to make it easier for patients to treat at home.
Our care team celebrates the more than 50,000 DaVita patients who are alive today because of transplants, and we aspire to ensure that even more patients on the waitlist receive a transplant. We are excited that the Administration is focused on improving the organ supply so that more patients can get a kidney transplant.’
Appeals Court Judges Appear Skeptical About ObamaCare’s Future
If its line of questioning serves as a barometer, a three-judge panel of the US Fifth Circuit Court of Appeals here seemed to be more favorably inclined toward the arguments of a group of 18 Republican states and two individuals seeking to invalidate the Affordable Care Act (ACA) than to those bent on defending the law.
“I think the plaintiffs had a better day than the defendants,” Josh Blackman, an associate professor of law at the South Texas College of Law, Houston, told Medscape Medical News.
“I think they found that the plaintiffs had standing,” said Blackman, who attended the arguments. The judges also seemed to believe the plaintiffs have been injured by the ACA, and that the individual mandate still demanded that people buy health insurance, even though Congress has eliminated the penalty, he said.
“Short news is it went very badly,” said Ian Millhiser, a senior fellow at the liberal-leaning Center for American Progress, on Twitter, after attending the hearing.
Ian Millhiser@imillhiser
Leaving the Fifth Circuit Obamacare case now. Short news is it went very badly. The two Republican judges appear determined to strike Obamacare.There is a chance they will be too embarrassed to do so, but don’t bet on it.
“The two Republican judges appear determined to strike Obamacare,” he said, adding, “There is a chance they will be too embarrassed to do so, but don’t bet on it.”
At the outset, Judge Jennifer Walker Elrod asked Samuel Siegel, a lawyer with the California Department of Justice representing the 20 states and Washington, DC, who are defending the ACA, “If you no longer have the tax, why isn’t [the ACA] unconstitutional?”
Only two of the three judges on the panel asked questions during the 1-hour-and-46-minute hearing — Elrod, appointed by President George W. Bush in 2007, and Kurt Engelhardt, appointed by President Donald J. Trump in 2018. Carolyn Dineen King, appointed by President Jimmy Carter in 1979, did not ask a single question.
The defendants — led by California — were first to argue. They were given 45 minutes to make their case that District Court Judge Reed O’Connor in Texas had erred in December when he ruled that the ACA should be struck downbecause Congress had eliminated the penalty associated with the requirement that individuals buy health insurance.
Essentially, said Judge O’Connor, the mandate could not be severed from the rest of the ACA. O’Connor did not grant the plaintiffs’ request that the ACA be halted while the case made its way through the courts.
The plaintiffs — led by Texas Solicitor General Kyle Hawkins — also had 45 minutes before the appellate court judges.
Is the ACA Now a “Three-Legged Stool?”
Both Judges Elrod and Engelhardt interrupted Siegel several times while arguing for the ACA to ask him to explain why California and the other states had the standing to defend the federal law. Siegel said that if the law were to be struck down it would cost the defendants hundreds of billions of dollars.
The two judges seemed intent on getting both sides to explain why Congress would have eliminated the penalty that went along with the individual mandate but left the rest of the law standing. The plaintiffs contend that the law could not be severed into parts, that it lived or died with the mandate and its penalty.
When asked to assess congressional intent, Hawkins said, “I’m not in the position to psychoanalyze Congress.” But he said the US Supreme Court had already settled the question, ruling in King v Burwell that the ACA was like a three-legged stool without the penalty. And, he said, even without the penalty, the individual mandate remained part of the law, which he called “a command to buy insurance.”
Douglas Letter, the general counsel to the US House of Representatives, arguing in defense of the ACA, said the opposite: that the Supreme Court had determined in NFIB v Sebelius that the ACA presented a choice of buying health insurance or facing a penalty. Without the penalty, “The choice is still there,” said Letter, adding that individuals could choose to maintain insurance or not.
“We know definitively that ‘shall’ in this provision does not mean must,” Letter said.
Engelhardt disagreed and said that Congress perhaps should have revised the ACA after the penalty was removed. He also asked Letter why the Senate was not also a party to the defense of the ACA. “They’re sort of the 800-pound gorilla not in the room,” Engelhardt said.
What’s Next?
The judges are not expected to rule for several months, and will be addressing several issues, including whether the Democratic states and the House of Representatives have proper standing to defend the law and whether the plaintiffs have standing to challenge the law.
They also will address whether the individual mandate is still constitutional, and if the mandate is ruled unconstitutional, whether it can be severed from the rest of the ACA, or, on the other hand, whether other provisions of the ACA also must be invalidated, according to the Kaiser Family Foundation.
The court could dismiss the appeal and vacate O’Connor’s judgment, “in which case there wouldn’t be any decision in the case at all,” Timothy S. Jost, professor emeritus at the Washington and Lee University School of Law in Lexington, Virginia, told Medscape Medical News ahead of today’s hearing.
At the hearing, Texas’ Hawkins said it was wrong to say the plaintiffs were trying to strike the law. “There’s an erasure fallacy,” he told the judges. “We’re not asking the court to erase anything.”
Still, O’Connor did say in his ruling that the ACA was unconstitutional. The Trump administration announced in March that it would not defend the law, but said it would continue to enforce the ACA. August E. Flentje, a US Department of Justice lawyer, reiterated that position at the Fifth District hearing today.
But, in a briefing before the hearing, the administration argued that, if ultimately the law is ruled unconstitutional, it should only be struck down in the states seeking to overturn the law. Any ultimate judgement “should not declare a provision unlawful if it doesn’t impact the plaintiff,” Flentje said.
Douglas Letter, for the defendants, was agog. “The DOJ position makes no sense,” he said, noting, for instance, that that would mean that the US Food and Drug Administration — which is required to speed biosimilar drugs to market under the ACA — would approve drugs in California, but not Arizona.
Elrod pressed the point by asking Flentje, “What is the government planning to do?” if the ACA is halted.
“A lot of this has to get sorted out — it’s complicated,” he said.
Despite the outcome of today’s hearings, the case is still ultimately expected to go before the Supreme Court again, according to multiple legal experts.
Advocates: The Stakes Are Astronomical
Shortly after the hearing ended, California Attorney General Xavier Becerra issued a statement predicting disaster for American healthcare if the appeals court agrees that the ACA is unconstitutional. If that happens, “Millions of Americans could be forced to delay, skip, or forego potentially life-saving healthcare,” he said.
“Our state coalition made it clear: on top of risking lives, gutting the law would sow chaos in our entire healthcare system,” Becerra said, vowing to “fight the Trump Administration tooth and nail.”
Physicians, consumer and patient advocates, and healthcare groups have voiced their support of the law through friend-of-the-court briefs, starting in June 2018, when the American Medical Association, the American College of Physicians, the American Academy of Family Physicians, the American Academy of Pediatrics, and the American Academy of Child and Adolescent Psychiatry joined together in a brief.
Other organizations have also voiced their support for the ACA through amicus briefs, including: the American Hospital Association, the Federation of American Hospitals, the Catholic Health Association of the United States, the Association of American Medical Colleges, Americas Health Insurance Plans, the Disability Rights Education and Defense Fund, the Blue Cross Blue Shield Association, Families USA, AARP, the Children’s Partnership, 483 federally recognized tribal nations, and 35 cities, counties, and towns.
A coalition led by the American Cancer Society, and including the American Diabetes Association, the American Heart Association, the American Lung Association, the Crohn’s & Colitis Foundation, the Cystic Fibrosis Foundation, the Epilepsy Foundation, the Hemophilia Federation of America, the Leukemia & Lymphoma Society, the March of Dimes, the National Alliance on Mental Illness, the National Coalition for Cancer Survivorship, the National Hemophilia Foundation, the National Multiple Sclerosis Society, and The Kennedy Forum also filed an amicus brief and issued a joint statement ahead of the hearing.
“If allowed to stand, the lower court’s ruling would once again mean people could be charged more or denied coverage based on their health history,” said the statement. “Insurance plans could impose arbitrary annual and lifetime limits on patients’ coverage and could exclude whole categories of care — like prescription drugs — from their plans,” they said, adding that striking the law from the books would jeopardize tax credits used by 8 million Americans to buy health insurance on the individual market.
Millions more could be dropped from Medicaid, the coalition stated. In total, the groups said that some 27 million people could lose health insurance — a figure they said was calculated by the Congressional Budget Office.
The Kaiser Family Foundation estimated that 19 million people could lose insurance. Also at stake: requiring private insurance, Medicare, and Medicaid expansion coverage of preventive services with no cost sharing, and a phase-out of the Medicare prescription drug “doughnut hole” coverage gap.
“All of these provisions could be overturned if the trial court’s decision is upheld, and it would be enormously complex to disentangle them from the overall health care system,” Kaiser said.
The Urban Institute estimated that if the ACA were overturned, the number of uninsured would increase by 65% — 20 million people; state spending on Medicaid/Children’s Health Insurance Program (CHIP) would fall by $9.6 billion — and that uncompensated care would rise by $50.2 billion, an increase of 82%.
Insurer Clover Health launches drug company
Medicare Advantage insurer Clover Health is launching a business to develop more effective medicines for elderly patients with chronic diseases.
The subsidiary, called Clover Therapeutics, hopes to involve its members in voluntary research programs to better understand the genetic drivers of their chronic conditions so it can create tailored therapies.
“We think we can do more than just coordinating care,” said Cheng Zhang, who is heading up the new business. “If we are able to elevate the standard of care, meaning developing new medicines and better, more effective medicines, that could really result in even better outcomes.”
Marcel van der Brug, the chief scientific officer for Clover Therapeutics, said the drugs on the market today target a specific type of age-related macular degeneration that affects just a sliver of patients with the disease.
“So there’s a huge unmet need there,” van der Brug said.
That plus the fact that ocular diseases are often driven by genetic factors give Clover a chance to understand the molecular drivers of the disease and create a novel drug to reach the broader population that doesn’t respond to what’s available, he explained.
Clover is partnering with biotech company Genentech, which is owned by Roche, to research and develop the drugs. Zhang explained that Clover will be involved in designing the clinical studies, collecting and analyzing data and making its own discoveries.
Clover has previously showed an interest in expanding its members’ access to targeted medicines. Last year, the insurer announced it would start providing genetic testing to patients in their homes to make sure they receive the most-effective drug combinations and avoid averse effects.
Just a few months ago, Clover announced plans to lay off about a quarter of its workforce to make room for experts in clinical care and insurance, according to several media reports.
Clover has not set any goals related to how many drugs it will produce. Zhang said the company’s focus is not on developing drugs to produce revenue. The focus is on patient outcomes.
“This is really about how do we better care for our patients, and we really think that research can be a form of clinical care. It can be integrated as such,” Zhang said. “As an organization, what we are focusing on is trying to maximize the benefits of research for patients as well as families, as well as others like them.”
Johnson & Johnson Wins New Trial In Talc Case
An appeals court has ruled that a new trial is in order for Johnson & Johnson JNJ 0.14% after a jury ordered the company to pay $417 million to a woman who claims the company’s baby powder caused cancer, according to Bloomberg.
While there was was sufficient evidence to uphold the jury’s finding that Johnson & Johnson failed to warn Eva Echeverria about the health risks of using the baby powder, the court said there is conflicting evidence about the product’s cancer links, according to Wednesday’s report.
In May, Johnson & Johnson was ordered by a jury to pay $300 million in punitive damages to Donna Olson, who has mesothelioma.
Johnson & Johnson faces more than 14,000 claims that its talc powder caused ovarian cancer and mesothelioma, a rare cancer linked to asbestos exposure.
Trials over the cancer claims tied to Johnson & Johnson’s talc-based powders first began in 2016.
MacroGenics teams up with I-Mab to develop cancer candidate
MacroGenics (NASDAQ:MGNX) inks an exclusive license and collaboration agreement with I-Mab Biopharma to develop and commercialize enoblituzumab in China, Hong Kong, Macau and Taiwan.
I-Mab will lead regional trials and will participate in global studies conducted by MGNX. A Phase 2 trial evaluating the combination of enoblituzumab and PD-1 inhibitor MGA012 in head and neck cancer should launch later this year.
Under the terms of the agreement, MGNX will receive $15M upfront, up to $135M in milestones and tiered double-digit royalties on net sales in the territory.
Enoblituzumab is an anti-B7-H3 monoclonal antibody that incorporates MGNX’s proprietary Fc Optimization technology platform.
Shares are up 2% after hours.
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