September - December 2019
In Stunning Twist, FDA Approves Sarepta’s Duchenne Drug It Rejected
The Food and Drug Administration approved Sarepta’s Duchenne muscular dystrophy therapy Vyondys 53, four months after rejecting the drug over safety concerns. The decision comes as a major surprise, as Sarepta had given no prior hints that it had resubmitted an application to the agency. Reversing a rejection so quickly is highly unusual. A type of nucleic acid therapy, Vyondys 53 is now conditionally cleared for the roughly 8% of patients with Duchenne who are “amenable” to exon 53 skipping, the mechanism by which the drug works. The FDA’s rejection of Vyondys 53 had stirred speculation the agency had done so for political reasons tied to the controversy over Exondys 51.
New Drugs for Sickle Cell Excite but, at ASH, a Recognition That More Is Needed
For decades, a repurposed cancer drug was the only treatment option for the tens of thousands of Americans who suffer from sickle cell disease, an inherited disorder that warps red blood cells to potentially devastating effects. That’s since changed, following a Food and Drug Administration approval for Endari in 2017 and then, just last month, for two new drugs from Novartis and Global Blood Therapeutics. The flurry of approvals, along with advancing development for nearly 20 other experimental therapies, is a dramatic change for a sickle cell disease community that hasn’t always been a focus for drug developers. The disease more commonly affects black and Hispanic people. Many of those affected are less well off and are covered by government insurance. https://www.biopharmadive.com/news/sickle-cell-new-drugs-excite-more-needed-ash/568690/
Hospital Groups Sue to Block Price-Transparency Rule
Hospital groups sued to block a Trump administration rule forcing them to disclose secret rates, for the first time laying out the industry’s legal strategy for defeating the president’s central health-policy initiative. The lawsuit filed Wednesday says the rule compelling the hospitals to publish their negotiated rates with insurers violates the First Amendment and goes beyond the statutory intent of the Affordable Care Act. The groups say the disclosure under the rule would be compelled speech in violation of the First Amendment. They are asking for an expedited decision, saying hospitals could otherwise spend needless time and resources preparing for a rule that may be invalidated by the court. https://www.wsj.com/articles/hospital-groups-sue-to-block-price-transparency-rule-11575460685
FTC Puts the Brakes on Illumina’s $1.2B Offer for DNA Sequencing Rival PacBio
The Federal Trade Commission has moved to block Illumina’s $1.2 billion takeover of its emerging rival, Pacific Biosciences, saying the DNA sequencing giant would substantially be harming competition. The commission also gave its staff clearance to seek temporary restraining orders or federal injunctions, if necessary, to maintain the current business while its complaint is heard by an administrative law judge in a formal hearing. “When a monopolist buys a potential rival, it can harm competition,” said Gail Levine, deputy director of the FTC’s Bureau of Competition, in a statement. “These deals help monopolists maintain power. That’s why we’re challenging this acquisition.” The FTC has claimed that the acquisition would reduce the combined company’s incentives to develop new products and that Illumina and PacBio’s rivalry drives them to innovate. https://www.fiercebiotech.com/medtech/ftc-blocks-illumina-s-1-2b-offer-for-dna-sequencing-rival-pacbio
SABCS: 10-Year Study Shows Targeted Radiation Can Be as Effective as Whole-Breast Doses Against Cancer
A decade-long study found patients with early breast cancer may be spared radiation procedures that span the whole breast, and that shorter and less invasive courses of treatment aimed at portions of the breast may be just as effective. After surgery to remove the tumor, patients who received accelerated partial breast irradiation saw similar rates of recurrence to those who received whole breast irradiation, according to the data presented at the San Antonio Breast Cancer Symposium. The study also found that it was unable to reject the idea that lowerdose radiation therapy was less effective compared to whole-breast treatments, with little difference between its two study arms. https://www.fiercebiotech.com/medtech/sabcs-10-year-study-shows-targeted-radiation-can-be-as-effective-as-wholebreast-doses
CVS, Rite Aid Join Lawsuit Over Diabetes Drug Overcharging
CVS Health and Rite Aid joined other pharmacy chains Monday in a lawsuit accusing three drugmakers of squeezing out generic competitors to protect their sales of a diabetes medication, resulting in $2.8 billion in excessive spending. The lawsuit details a series of events that allegedly impeded competitors via notorious “patent games” that policymakers aim to ban. The pharmacy chains allege Assertio Therapeutics paid Lupin Pharmaceuticals to delay its generic version of Glumetza, the extended-release iteration of a drug that has been used to treat Type 2 diabetes since 2002. While Lupin’s generic could have entered the market in 2009, the companies settled a related patent lawsuit and kept the competition at bay until 2016.
UPS Drone Makes First Home Prescription Deliveries for CVS
United Parcel Service Inc. Flight Forward drones have flown prescription medications to the front lawn of a private home and to a retirement center, the UPS unit’s first revenue-generating deliveries for drugstore chain CVS Health Corp. UPS and CVS said on Tuesday the deliveries were the first of their kind under an program approved by the U.S. Federal Aviation Administration (FAA). Regulators are still hammering out rules for how the unmanned winged vehicles will operate in U.S. airspace and guidelines are expected in 2021. UPS became the first company to win the broadest FAA certification to operate a drone airline. That permits Flight Forward to collect payment for drone deliveries and to fly as many drones supported by as many operators as necessary to meet customer demand. https://www.reuters.com/article/us-ups-drones/ups-drone-makes-first-home-prescription-deliveries-for-cvsidUSKBN1XF2JC
Novartis Pivots Shanghai R&D Site From Early Discover to Development
Novartis is calling it curtains on early drug discovery at its R&D site in Shanghai in a companywide move to “rebalance” its discovery and early development efforts. The company plans to add more than 300 new jobs in the coming years but will be bidding adieu to about 150 early discovery personnel. “Our need to better resource early drug development globally, combined with important changes to the drug development and commercialization landscape in China, have converged on the decision to pivot from drug discovery in Shanghai to early drug development,” said Jay Bradner, M.D., president of the Novartis Institutes for BioMedical Research (NIBR). https://www.fiercebiotech.com/biotech/novartis-pivots-shanghai-r-d-site-from-early-discovery-to-development
Google’s Health Care Ambitions Now Involve Thousands of Patient Records
Google announced a partnership with a large U.S. health care system aimed at modernizing its information system and providing new tools for doctors, in the tech giant’s latest foray into the health industry. Google is providing cloud computing services to Ascension, which operates health centers in 21 states, mostly across the South and Midwest. It is also testing the use of artificial intelligence to examine health records and find patterns that Google says might help doctors and other providers. Both companies stressed that their deal is compliant with federal health-privacy law. Unlike most of the data Google collects on individuals, health data is strictly regulated by the federal government. Health care providers are increasingly interested in using data to help manage care and keep patients healthy.
Genome Sequencing in Newborns Raises Ethical Issues
In 2014, the National Institutes of Health funded four projects to study the benefits and risks of genomic sequencing for newborns. During the study, the research team felt moral distress about not being able to disclose the information because it wasn’t related to a childhood disease, so they approached their institutional review board and asked for permission to disclose it and then told the baby’s parents. Ultimately, the study protocol was modified to require all participating families to agree to receive information about adult-onset conditions, too. However, Dr. Lainie Friedman Ross of the MacLean Center addressed that sequencing all or large parts of a baby’s genome at birth could reveal genetic variations that increase risk for conditions that occur in childhood or not until adulthood. The conditions could be benign or ultimately be untreatable later. https://www.reuters.com/article/us-health-newborns-genomics-ethics/genome-sequencing-in-newborns-raises-ethicalissues-idUSKBN1XN2TR
Trump Administration Sues Gilead Over HIV Drug Patents
The U.S. government on Wednesday accused Gilead of illegally profiting from taxpayer-funded HIV research, suing the biotech in federal district court for infringing on patents tied to the preventive use of two HIV drugs. The lawsuit escalates a dispute between Gilead and the Department of Health and Human Services, which holds four patents for research done in the 2000s by the Centers for Disease Control and Prevention on preexposure prophylaxis HIV treatment. Gilead has refused to license those patents from HHS, the agency said, and in August contested their validity by requesting the U.S. Patent Office review all four patents. Gilead sells two drugs, Truvada and Descovy, for PrEP, expanding the use of which is a major component of the Trump administration’s plans to reduce new HIV infections in the U.S.
China Approves Alzheimer’s Drug, Inviting Fresh Debate in Field With Few Successes
In a decision likely to stir scientific debate across the world, China’s health regulator conditionally approved a new Alzheimer’s drug for patients with mild-to-moderate forms of the neurodegenerative disease. The drug, which was developed by the little-known Shanghai Green Valley Pharmaceuticals, will be available throughout China by the end of the year, according to the company and an English statement posted on the website of China’s National Medical Products Administration. No new Alzheimer’s drug has won approval since 2003 and drugmaker efforts to change that have consistently come up short, making the NMPA’s decision notable. But it’s not clear how effective Green Valley’s drug is, with limited information about the positive trial results reported by the company last year. https://www.biopharmadive.com/news/china-alzheimers-drug-oligomannate-approval-green-valley/566540/
Merck to Lay Off 500 US-Based Commercial Employees
Merck & Co. plans to lay off approximately 500 U.S.-based employees working on sales and commercial teams, the company disclosed Thursday in a regulatory filing and confirmed Friday to BioPharma Dive. Affected employees, who are spread out across the country, will be laid off on Jan. 3, 2020 with separation packages, according to a company statement. Merck has also reduced its U.S. workforce by 40% in the past decade to 25,400 workers at the end of 2018. These newest job cuts appear set to further lower that figure, although a Merck spokesperson noted the pharma is hiring for U.S. oncology-focused jobs. “This is not a new restructuring effort,” Merck spokesperson Pamela Eisele said in a statement. Instead, the layoffs are part of “ongoing company-wide efforts to sharpen Merck’s focus” on R&D growth opportunities.
Johnson & Johnson’s Legal Challenges Mount
Johnson & Johnson, facing lawsuits from more than 100,000 plaintiffs over its product safety and marketing tactics, has taken the aggressive strategy of battling many of the cases in court, and is losing. Juries and judges have ordered the health-products giant to pay billions of dollars in several recent trials over claims that J&J’s signature baby powder and certain drugs and medical devices injured people, and that its marketing practices fueled the opioid-addiction epidemic. The number of talc-lawsuit plaintiffs surged to 15,500 as of June 30, from 1,400 in early 2016, the Journal analysis found. Plaintiffs in personal-injury lawsuits over J&J’s pelvic mesh devices for women have declined from a peak of more than 55,000 pending in 2017 but still number about 24,800. https://www.wsj.com/articles/johnson-johnsons-legal-challenges-mount-11571055242
Bayer to Back 11 International Startups in Various Digital Health Enterprises
Bayer has signed onto sprawling new collaborations with 11 digital health startups spanning areas such as oncology, ophthalmology, pulmonology, radiology, digital therapeutics and cardiovascular health. The program also paves the way for longer-term collaborations, including commercial development support to bring the digital health products to market, said Zsuzsanna Varga, head of Bayer’s G4A program, which is divided into two different pathways. The “Growth Track” grants early-stage startups €75,000 ($82,700 U.S.) for co-creating products, plus co-working space and mentoring at Bayer’s pharmaceuticals division headquarters in Berlin. Meanwhile, the “Advance Track” includes startups looking to co-create and execute commercial deals and includes incremental milestone-based payments.
Purdue Pharma, Maker of OxyContin, Files for Bankruptcy
Purdue Pharma, maker of OxyContin, the drug widely seen as igniting the opioid crisis, filed for Chapter 11 bankruptcy on Sunday night, a move at the center of the company’s efforts to shield itself and its owners from more than 2,600 federal and state lawsuits. The terms of the filing are expected to be fiercely contested by a group of states that have refused to settle with Purdue and are intent on pursuing the company’s owners, the Sacklers, considered one of the wealthiest families in the United States. Restructuring the company through bankruptcy was at the heart of a tentative settlement agreement reached between the company and thousands of cities and counties that have sued it in federal court for its role in the opioid epidemic.
Roivant Offloads Biotech Stakes in $3B Alliance With Japanese Pharma
Sumitomo Dainippon Pharma will pay Roivant Sciences $3 billion for its stake in five of the holding company’s subsidiaries, and take a more than 10% stake in the parent company founded by unorthodox CEO Vivek Ramaswamy. Through the alliance, Sumitomo Dainippon additionally gains the option to acquire Roivant’s stake in six more subsidiaries with up to 25 pipeline projects. The Japanese pharma is looking to replace lost revenue from the looming expiration of U.S. patent expiration on its biggest seller, the antidepressant Latuda, which had sales of more than $1.7 billion in the company’s 2018 fiscal year. https://www.biopharmadive.com/news/roivant-deal-sumitomo-dainippon-3-billion-ramaswamy/562401/
Regulatory Barriers Limit Alternative State Drug Payment Models
More states are turning to alternative payment models for prescription drugs covered under Medicaid, as they seek to balance increasing costs with public health goals. Despite regulatory and implementation barriers that still impede the spread of outside-the-box approaches, Louisiana’s “Netflix” model approach has captured many headlines. The state reached a deal with a subsidiary of drugmaker Gilead in which the state will receive an unlimited supply of its hepatitis C drug Harvoni for a fixed sum each year for five years. Other ideas build incentives into contracts to reward for positive outcomes. Oklahoma, Michigan and Colorado have negotiated outcomes-based contracts with drug manufacturers, in some way reimbursing the drugmaker for how well the drug works. https://www.biopharmadive.com/news/regulatory-barriers-limit-alternative-state-drug-payment-models/563204/
Israel Prepares to Unleash AI on Health Care
Israel is becoming a testing ground for the power of artificial intelligence to improve health care. Digital medical records for the vast majority of Israelis are currently stored in databases maintained by the handful of semipublic HMOs that provide most health care in Israel. While the biggest health-maintenance organizations already leverage their records in partnerships with private companies to develop technology for more advanced health care, Israel’s government wants to take such efforts to a new level. And the greater goals, officials say, based largely on the promise of AI technology, are to make health care less expensive, more effective and better tailored to individuals everywhere. https://www.wsj.com/articles/israel-prepares-to-unleash-ai-on-health-care-11568599261