Genmab Get Euro OK for Front Line Multiple Myeloma Med

Genmab A/S (Nasdaq Copenhagen: GEN) announced today that the European Commission (EC) has granted marketing authorization for DARZALEX(daratumumab) in combination with bortezomib, melphalan and prednisone (VMP), for the treatment of adult patients with newly diagnosed multiple myeloma who are ineligible for autologous stem cell transplant (ASCT).

The EC approval follows a positive opinion issued for DARZALEX by the CHMP of the European Medicines Agency (EMA) in July 2018. In August 2012, Genmab granted Janssen Biotech, Inc. (Janssen) an exclusive worldwide license to develop, manufacture and commercialize daratumumab.

Genmab will receive a milestone payment of USD 13 million from Janssen in connection with the first commercial sales of DARZALEX under the expanded label. The sales are expected to occur quickly after the approval. This milestone payment was included in the financial guidance issued by Genmab originally on February 21, 2018 and then reiterated in subsequent quarterly financial reports, most recently on August 8, 2018, and as such there is no change to the company’s financial guidance following this approval.

‘Approved in this indication in the U.S. since early May, DARZALEX in combination with bortezomib, melphalan and prednisone will now become an option for newly diagnosed multiple myeloma patients in Europe,’ said Jan van de Winkel, Ph.D., Chief Executive Officer of Genmab. ‘We are very pleased that many more patients in need will have the opportunity for treatment with this regimen and we look forward to seeing this combination launched in Europe.’

The positive opinion of the CHMP was based on data from the Phase III ALCYONE (MMY3007) study that showed a reduction of the risk of disease progression or death by 50 percent (Hazard Ratio [HR] = 0.50; 95 percent CI [0.38-0.65], p

https://www.marketscreener.com/GENMAB-1412914/news/Genmab-Announces-European-Marketing-Authorization-for-DARZALEX-for-Front-Line-Multiple-Myeloma-27200908/

Inventiva achieves 3 key milestones with lead product

Inventiva S.A. (‘Inventiva’ or the ‘Company’), a biopharmaceutical company developing innovative therapies in nonalcoholic steatohepatitis (NASH), systemic sclerosis (SSc) and mucopolysaccharidosis (MPS), today announced the successful completion of three key milestones in the development of its lead product, lanifibranor.

New patent granted by the USPTO protecting the use of lanifibranor in numerous fibrotic diseases

On August 21, 2018, the United States Patent and Trademark Office (USPTO) granted a new patent protecting until June 2035 the therapeutic use of lanifibranor in the treatment of various fibrotic conditions, including NASH and SSc.

This patent strengthens and extends in the United States the term of protection of lanifibranor derived from the New Chemical Entity (NCE) patent expiring in December 2031 (this expiration date includes a possible five-year extension to compensate for regulatory delays in obtaining the marketing approval).

Enrolment of the first patient in the United States Phase II study for the treatment of NAFLD in patients with type 2 diabetes

Following the agreement with the University of Florida to conduct a Phase II study in the United States with lanifibranor for the treatment of non-alcoholic fatty liver disease (NAFLD) in patients with type 2 diabetes, the first of 64 patients for this study has been enrolled in August 2018. The recruitment of the other patients continues in accordance with the planned schedule and top line results are expected in early 2020.

The overall objective of the study , led by Dr Kenneth Cusi, Head of the Department of Endocrinology, Diabetes & Metabolism in the Department of Medicine at the University of Florida at Gainesville, is to measure the metabolic improvements induced by lanifibranor, as well as its effects on hepatic steatosis in patients with type 2 diabetes and NAFLD. In addition, this study will examine the impact of lanifibranor on fibrosis using the latest imaging and biomarker technologies.

FDA approval of the IND application enabling to launch the clinical development plan in the United States.

https://www.marketscreener.com/INVENTIVA-34031212/news/Inventiva-advances-in-the-development-of-lanifibranor-with-the-achievement-of-three-key-milestones-27200927/

Sanofi approved in Europe for adults blood clotting disorder med

The European Commission has granted marketing authorization for Cablivi™ (caplacizumab) for the treatment of adults experiencing an episode of acquired thrombotic thrombocytopenic purpura (aTTP), a rare blood-clotting disorder. Cablivi is the first therapeutic specifically indicated for the treatment of aTTP.

aTTP is a life-threatening, autoimmune-based blood clotting disorder characterized by extensive clot formation in small blood vessels throughout the body, leading to severe thrombocytopenia (very low platelet count), microangiopathic hemolytic anemia (loss of red blood cells through destruction), ischemia (restricted blood supply to parts of the body) and widespread organ damage especially in the brain and heart.

Despite the current standard-of-care treatment, consisting of daily plasma exchange (PEX) and immunosuppression, episodes of aTTP are still associated with a mortality rate of up to 20%, with most deaths occurring within 30 days of diagnosisⁱ.

“aTTP is a devastating disease. Many patients undergoing current standard-of-care treatment continue to be at risk of developing acute thrombotic complications, including stroke and heart attack, recurrence of the disease, lack of treatment response and death,” said Marie Scully, M.D, professor of hematology at University College London Hospitals.  “The approval of Cablivi provides an important addition to the standard-of-care treatment for patients with aTTP in Europe because it can significantly reduce time to platelet count normalization and induce a clinically meaningful reduction in recurrences.”

Cablivi was developed by Ablynx, a Sanofi company. Sanofi Genzyme, the specialty care global business unit of Sanofi, will work with relevant local authorities to make Cablivi available to patients in need in countries across Europe.

Cablivi is the company’s first Nanobody^®-based medicine to receive approval and the first newly approved product that will be part of Sanofi Genzyme’s Rare Blood Disorders franchise. Earlier this year, Sanofi acquired Bioverativ which has treatments for hemophilia A and B.

https://www.marketscreener.com/SANOFI-4698/news/Sanofi-Cablivi-trade-caplacizumab-approved-in-Europe-for-adults-with-acquired-thrombotic-thromb-27201597/

FDA Accepts Bristol-Myers Application for Leukemia Med in Pediatrics

Bristol-Myers Squibb Company (NYSE: BMY) today announced that the U.S. Food and Drug Administration (FDA) accepted its supplemental Biologics License Application (sBLA) for Sprycel (dasatinib) in combination with chemotherapy for the treatment of pediatric patients with newly diagnosed Philadelphia chromosome-positive (Ph+) acute lymphoblastic leukemia (ALL).

The FDA action date is December 29, 2018.

‘Sprycel was first established as an important treatment option for appropriate pediatric patients last year, when it was approved for the treatment of children with Ph+ chronic myeloid leukemia,’ said Jeffrey Jackson, Ph.D., development lead, hematology, Bristol-Myers Squibb. ‘This latest milestone in Ph+ ALL reinforces our commitment to researching the potential of Sprycel in different types of pediatric leukemia and to providing this vulnerable population with access to potential new therapies.’

The application is based on data from CA180-372 (NCT01460160), an ongoing Phase 2 trial evaluating the addition of Sprycel to a chemotherapy regimen modeled on a Berlin-Frankfurt-Munster high-risk backbone in pediatric patients with newly diagnosed Ph+ ALL.

About Sprycel

Sprycel first received FDA approval in 2006 for the treatment of adults with Philadelphiachromosome-positive (Ph+) chronic myeloid leukemia (CML) in chronic phase (CP) who are resistant or intolerant to prior therapy including imatinib. At that time, Sprycel also received FDA approval for adults with Ph+ acute lymphoblastic leukemia (ALL) who are resistant or intolerant to prior therapy. Sprycel is approved and marketed for these indications in more than 60 countries.

Sprycel is also an FDA-approved treatment for adults with newly diagnosed Ph+ CML-CP and is approved for this indication in more than 50 countries.

Both the FDA and the European Commission approved the expansion of Sprycel’s indication to include pediatric patients with Ph+ CML-CP in November 2017 and July 2018.

https://www.marketscreener.com/BRISTOL-MYERS-SQUIBB-COMP-11877/news/Bristol-Myers-Squibb-U-S-Food-and-Drug-Administration-Accepts-Bristol-Myers-Squibb-s-Application-27200891/

Dechra Pharmaceuticals says to implement hard Brexit plan

Veterinary drugs producer Dechra Pharmaceuticals said on Monday it was moving ahead with plans for a hard Brexit, stressing the financial impact should be “immaterial” but spooking investors worried about revenue and operations after next year’s split.

Shares of the company plunged around 20 percent to a more than six-month low to 2,454 pence in early trading on the London Stock Exchange.

“Our primary focus is on addressing Brexit risk in our supply chain,” the company said, listing costs worth a total of just 2 million pounds in relation to the plans.

“This includes transferring UK registered Marketing Authorisations for products that are sold in the EU to an EU entity and duplication of product release testing for products that are transferred between the UK and the EU,” it said.

Dechra shares had risen almost 54 percent till Friday’s close, and analysts and traders said the sell-off on Monday looked overdone.

The company listed Brexit along with U.S. sanctions on Iran and the European Union challenging the legality of the UK Control Foreign Company (CFC) tax legislation as three emerging geopolitical risks to its business.

Results for the FTSE 250-listed firm showed revenue rose 13.9 percent to 407.1 million pounds at constant currency for the year-ended June 30. Revenue from its largest EU Pharmaceuticals segment rose 11.4 percent.

https://www.marketscreener.com/DECHRA-PHARMACEUTICALS-4003059/news/Dechra-Pharmaceuticals-Pharma-says-to-implement-hard-Brexit-plan-27200653/

5 Kavanaugh rulings that will loom large this week

President Donald Trump’s second Supreme Court nominee Brett Kavanaugh begins his confirmation hearings September 4. While abortion and Roe v. Wade are in the spotlight, his positions on other key healthcare issues will come into play, including Obamacare.

Kavanaugh’s record includes significant writings on the Affordable Care Act, Medicare, contraception and abortion, but they are nuanced—offering a difficult task for Democrats on the Judiciary Committee who will try to pin him down on how he might rule from the Supreme Court bench.

Below are five cases that offer insights into his thinking on controversial healthcare questions.

1) Obamacare Appeal No. 1: Seven-Sky v. Holder

Kavanaugh wrote the lengthy sole dissent in the 2011 federal appellate ruling that upheld the Affordable Care Act, but notably he did not indicate opposition to the law itself.

He questioned the precedent set by allowing Congress to penalize people for failing to comply with the federal mandate to buy insurance. He also opined that the mandate may show a new legislative approach for how Congress could design the safety net through the private sector, in which case he advised that the judiciary should stay out of Congress’ way. Conservative critics say Kavanaugh’s opinion laid the groundwork for the Supreme Court’s 2012 decision upholding the law.

“Privatized social services combined with mandatory-purchase requirements of the kind employed in the individual mandate provision of the Affordable Care Act might become a blueprint used by the Federal Government over the next generation to partially privatize the social safety net and government assistance programs and move, at least to some degree, away from the tax-and-government-benefit model that is common now,” he wrote. “Courts naturally should be very careful before interfering with the elected Branches’ determination to update how the National Government provides such assistance.”

2) Obamacare Appeal No. 2: Sissel v. HHS

This appeal argued that because the Senate replaced the House version of the ACA with its own language before passage, Congress violated the constitutional clause requiring any major tax legislation to originate in the House. Kavanaugh sided with the majority in upholding the ACA, but for a different reason than his colleagues. He wrote that Congress did not violate the clause in question; the other judges ruled that the so-called origination clause did not apply to the ACA.

It remains to be seen whether Kavanaugh can satisfy Democrats on his stance on the ACA coverage protections. Democrats who have met with him personally have pressed him on the issue have expressed varying degrees of skepticism.

“It is an issue that will affect millions of American families in really profound ways,” said Sen. Sheldon Whitehouse (D-R.I.), who sits on the Judiciary Committee. “And Kavanaugh being way too cute about his thoughts about it opens wide the prospect he will follow the Sessions path of inventing a constitutional problem with it.”

3) Abortion: Garza v. HHS

Abortion-rights groups point to this case from last October as being most indicative of Kavanaugh’s position on abortion and, potentially, Roe v. Wade.

Kavanaugh dissented from the majority decision to reverse an HHS order that barred an immigrant minor in federal custody from obtaining an abortion. In his opinion, Kavanaugh acknowledged the plaintiff’s right to abortion under Roe v. Wade, but argued the U.S. government shouldn’t be mandated to facilitate abortion on demand for undocumented immigrant minors in its custody. He said that a delay until the plaintiff found a U.S. sponsor didn’t qualify as “undue burden.”

Notably, he did not join another colleague in a dissent that argued an undocumented immigrant minor does not have the constitutional right to an abortion.

Republican Sens. Susan Collins (Maine) and Lisa Murkowski (Alaska), who support abortion rights, are facing the most intense pressure on this issue.

After meeting with Kavanaugh for more than two hours, Collins said that the nominee “expressed agreement with Chief Justice Roberts’ confirmation hearing statement that Roe is settled precedent and entitled to respect under principles of stare decisis.”

While legal experts consider Roe v. Wade safe for now, Planned Parenthood has counted 13 lawsuits involving strict abortion laws that could make it to the Supreme Court and analysts expect Kavanaugh would narrow the definition of what qualifies as an undue burden. This is enough to draw criticism from abortion rights advocates.

“The reality is that people in some states have to navigate nearly insurmountable barriers just to access care, including medically unnecessary waiting periods and the need to travel farther distances to get to clinics, which increases the cost if they have to pay for travel and child care,” said Dr. Willie Parker, board chair of Physicians for Reproductive Health.

4) Contraception: Priests for Life v. HHS

This religious liberty case upheld the ACA mandate that employers must offer coverage of contraception even if they object on religious grounds.

Kavanaugh dissented but, as in Garza v. HHS, took a more nuanced position than the other dissenters. He argued that the government “has a compelling interest in facilitating access to contraception” for employees of a religious organization, but stated the government should do so in the least restrictive way possible.

The insurer of these employees must continue to offer them contraceptive coverage, he wrote, but the employer should not be required to pay for it.

5) Medicare: Hall v. Sibelius

Kavanaugh sided with the majority in ruling against plaintiffs who didn’t want to be entitled to Medicare Part A because it would limit their ability to get private insurance.

Kavanaugh wrote that the law would not allow that.

“If you are 65 or older and sign up for Social Security, you are automatically entitled to Medicare Part A benefits,” he wrote. “You can decline those benefits. But you still remain entitled to them under the statute.”

http://www.modernhealthcare.com/article/20180901/NEWS/180839974

Pediatrics group urges parents to steer clear of popular FluMist in favor of shots

The American Academy of Pediatrics is recommending children be vaccinated with injectable flu vaccine for the coming season, rather than the nasal spray vaccine FluMist, unless a child will only be vaccinated if he or she can forgo a needle, or if a doctor runs out of flu shots.

“The AAP feels that the flu shot should be the primary vaccine choice for all children,” said Dr. Henry Bernstein, a pediatrician and an ex-officio member of the AAP’s committee on infectious diseases.

That advice puts the AAP’s annual flu vaccine recommendations slightly at odds with those of the Centers for Disease Control and Prevention, which state that any of the flu vaccines available for children could be used for the coming flu season.

Both, however, share an end goal: getting more children vaccinated.

Influenza can be deadly for children. In the past flu season, 180 children under the age of 18 died from the flu, making it the second most deadly flu season — after the 2009 H1N1 pandemic — since the CDC started recording pediatric flu deaths in the winter of 2005-2006.

The CDC recommends everyone over the age of 6 months be vaccinated every year against the flu, unless there is a medical reason to avoid the vaccine.

Yet only about 60 percent of children between the ages of 6 months and 17 years got vaccinated against flu in 2016-2017, the most recent year for which data are available. Roughly eight out of every 10 children who died from flu weren’t vaccinated, said Dr. Lisa Grohskopf, a medical officer in the CDC’s influenza division.

The differing advice from the CDC and the AAP on AstraZeneca’s FluMist could befuddle parents and pediatricians.

“There’s no question that ideally we would like for the CDC and the AAP to be completely harmonized” when it comes to recommendations, said Bernstein who is also a member of the Advisory Committee on Immunization Practices, which guides the CDC on vaccine decisions.

“Both groups are harmonized in wanting as many children to receive flu vaccine as possible each and every year,” said Bernstein. “When recommendations are not perfectly harmonized, it does pose the possibility for confusion.”

But any confusion might be mitigated by the fact that finding FluMist might not be easy this flu season.

The decision to once again recommend it — effectively giving doctors and pharmacists a go-head to use it again — was made by the ACIP in late February. By that point, though, many flu vaccine orders would already have been placed for the 2018-2019 season.

While dozens of lots of vaccine products made by Sanofi Pasteur, GlaxoSmithKline, and Seqirus have been given the green light for distribution by the Food and Drug Administration, no lots of FluMist had cleared that hurdle as of Aug. 30.

It has been a rocky few years for FluMist, which once was deemed more effective in children than injectable flu vaccine. But just after ACIP gave FluMist a rare preferential recommendation in 2014, performance problems came into view. By the 2016-2017 flu season, the CDC’s vaccine advisers recommended it not be used in the U.S. ACIP retained that position for the following flu season as well.

The problem was vaccine effectiveness studies that are done every year showed FluMist had not been offering much if any protection against the influenza A virus family H1N1.

It wasn’t clear why the vaccine’s performance was so poor in the U.S. during the 2013-2014 and 2015-2016 seasons. To make matters more confounding, other countries that use FluMist — Canada, Finland, and Britain among them — did not see the puzzling lack of effectiveness.

Unlike injectable flu vaccine, FluMist contains live viruses. The viruses in the vaccine, which is puffed up a nostril of the recipient, initiate the infection process, thereby activating an immune response. But the viruses in the vaccine are weakened and don’t induce illness.

In response to its U.S. performance problems, AstraZeneca reformulated the H1N1 portion of the vaccine. There is some evidence that suggests the updated vaccine may be more effective — although the company hasn’t been able to do studies in children to confirm that. H3N2 viruses have dominated in the last two flu seasons here.

“They provided some evidence that appeared promising that the new virus that’s going to go into the vaccine this season in the U.S. is a bit more fit,” said Grohskopf. “It is promising evidence that what is at least believed to be the root cause of the problem has been fixed.”

Bernstein said the AAP was not as convinced by the data as the CDC. He voted against recommending FluMist for the 2018-2019 season at the February ACIP meeting.

Another place the CDC and AAP advice diverges slightly relates to when children should be vaccinated.

The AAP recommendations, published Monday in the journal Pediatrics, suggest pediatricians should start urging parents to vaccinate their children as soon as flu vaccine becomes available. That can be as early as late July or August, which — depending on when flu activity picks up — is often months ahead of when children will face a flu threat. Flu season often peaks in January or February, and influenza B viruses, which are particularly hard on children, often circulate late in the winter.

There are some studies that have shown protection from flu vaccine starts to wear off as the season wears on.

The CDC’s recommendations, published Aug. 24, suggested people should be vaccinated by the end of October, when flu activity can start to tick up.

Grohskopf acknowledged the variability and unpredictability of influenza makes it tough to know when to advise people to get the vaccine. “What we can say is the best time to get vaccinated is probably a couple of weeks before flu starts circulating where you are. But we can never really tell people when that is,” she admitted.

One thing is clear though, she said. Parents of young children should start the vaccination process earlier. That’s because children under 8 years old who haven’t had at least two flu vaccines in their life need two doses of vaccine given at least four weeks apart. The second shot should be by the end of October, the CDC guidance said.

https://www.statnews.com/2018/09/03/flumist-vaccine-recommendations/