A drug whose inventors claim it can cure a rare form of blindness is to be one of the most expensive medicines ever sold at 0,000 (£630,000).
Luxturna is injected directly into the eye to address the root cause of visual impairment by replacing a defective gene in the retina. It is the first gene therapy to be approved for use in the US, and was given the go-ahead by the Federal Drug Administration last month. However, the price of the treatment has only just been revealed.
Spark Therapeutics, the company behind the treatment, had previously claimed the treatment was worth m, citing the cost of a lifetime of blindness in lost earnings and wages for caregivers. But the firm said it had settled for the lower price of 0,000, or 5,000 per eye. It will also offer ways to spread the cost to health insurers, which have expressed concern about their ability to cover the expense.
“We wanted to balance the value and the affordability concerns with a responsible price that would ensure access to patients,” said Spark Therapeutics’ chief executive, Jeff Marrazzo.
Luxturna is one of an emerging breed of gene therapies that differ from more established medicines administered over a period of time. Such treatments involve a one-off procedure to alter defective DNA, allowing the body to repair the problem itself.
They include a treatment for haemophilia and another for so-called “bubble baby” syndrome, where sufferers have to live in a sterile environment, which is to be offered on the NHS despite a £500,000 price tag.
The sky-high price of some drugs – and so-called price-gouging by drug firms – became an issue in the US presidential election after Martin Shkreli, a US hedge fund entrepreneur, bought the drug Daraprim, used in the treatment of Aids and cancer, and hiked its price from .50 to 0.
But the starting price of some new drugs has soared. Glybera, a gene therapy for a rare protein disorder, was launched in 2012 with a price tag of .2m. However, it was never approved in the US and was discontinued by manufacturers uniQure because of a lack of demand.
Gene therapy is not alone in commanding staggering sums, particularly when it comes to treatments for rare diseases. Soliris, a drug that treats a condition called paroxysmal nocturnal hemoglobinuria that attacks red blood cells, can cost up to 0,000 a year, while Elaprase, used in the treatment of Hunter syndrome, costs 0,000 a year.
Luxturna could potentially become available free on the NHS in the UK after being submitted for approval by the European Medicines Agency (EMA). A green light from the EMA is a prerequisite for approval by the National Institute for Health and Care Excellence, the UK’s healthcare costs watchdog.
In clinical trials, injections of Luxturna restored eyesight to people with severe visual impairment due to retinal dystrophy. Spark estimates that up to 2,000 people in the US suffer from the condition, with the number of sufferers rising to 6,000 when Europe and other markets where it could sell the treatment are included.
The company said it had agreed bespoke deals with US insurers, which cover the cost of most US prescriptions, and they will get a refund if the drug doesn’t work as expected.
Spark is also discussing a proposalfor insurers to pay for the drug in instalments over several years. The Philadelphia-based company said it would also pay transport costs not covered by insurance to help patients get access to treatment centres.
guardian.co.uk © Guardian News & Media Limited 2010
George HW Bush has died
George Herbert Walker Bush, the linchpin of an American political dynasty whose presidency saw the end of the Cold War and the close of an era of American bipartisanship that conflict fostered, has died. He was 94.
During his single term in the White House, the Berlin Wall fell, newly democratic states sprang up across Central and Eastern Europe, and the Soviet Union came to an end. And in the Middle East, the U.S. military launched its most successful offensive since World War II. For a time, Bush rode foreign policy triumphs to high popularity. But he saw his standing plunge during a 1990s recession and lost to Bill Clinton after one term.
On April 22nd President Bush was admitted to the Houston Methodist Hospital after contracting an infection that spread to his blood. He was said to have been responding to treatments and appeared to be recovering.
Court Orders White House to give Jim Acosta his hard pass back
Federal judge Timothy J. Kelly sided with CNN on Friday, ordering the White House to reinstate chief White House correspondent Jim Acosta’s press pass.
The ruling was an initial victory for CNN in its lawsuit against President Trump and several top aides.
The lawsuit alleges that CNN and Acosta’s First and Fifth Amendment rights are being violated by the suspension of Acosta’s press pass.
Kelly did not rule on the underlying case on Friday. But he granted CNN’s request for a temporary restraining order.
This result means that Acosta will have his access to the White House restored for at least a short period of time. The judge said while explaining his decision that he believes that CNN and Acosta are likely to prevail in the case overall.
CNN is also asking for “permanent relief,” meaning a declaration from the judge that Trump’s revocation of Acosta’s press pass was unconstitutional. This legal conclusion could protect other reporters from retaliation by the administration.
“The revocation of Acosta’s credentials is only the beginning,” CNN’s lawsuit alleged, pointing out that Trump has threatened to strip others’ press passes too.
That is one of the reasons why most of the country’s major news organizations have backed CNN’s lawsuit, turning this into an important test of press freedom.
But the judge will rule on all of that later. Further hearings are likely to take place in the next few weeks, according to CNN’s lawyers.
CNN sues President Trump for banning reporter Jim Acosta
CNN is filing a lawsuit against President Trump and several of his aides, seeking the immediate restoration of chief White House correspondent Jim Acosta’s access to the White House.
The lawsuit is a response to the White House’s suspension of Acosta’s press pass, known as a Secret Service “hard pass,” last week. The suit alleges that Acosta and CNN’s First and Fifth Amendment rights are being violated by the ban.
The suit is being filed in U.S. District Court in Washington, D.C. on Tuesday morning, a CNN spokeswoman confirmed.
Both CNN and Acosta are plaintiffs in the lawsuit. There are six defendants: Trump, chief of staff John Kelly, press secretary Sarah Sanders, deputy chief of staff for communications Bill Shine, Secret Service director Joseph Clancy, and the Secret Service officer who took Acosta’s hard pass away last Wednesday. The officer is identified as John Doe in the suit, pending his identification.
The six defendants are all named because of their roles in enforcing and announcing Acosta’s suspension.
Last Wednesday, shortly after Acosta was denied entry to the White House grounds, Sanders defended the unprecedented step by claiming that he had behaved inappropriately at a presidential news conference. CNN and numerous journalism advocacy groups rejected that assertion and said his pass should be reinstated.
On Friday, CNN sent a letter to the White House formally requesting the immediate reinstatement of Acosta’s pass and warning of a possible lawsuit, the network confirmed.
In a statement on Tuesday morning, CNN said it is seeking a preliminary injunction as soon as possible so that Acosta can return to the White House right away, and a ruling from the court preventing the White House from revoking Acosta’s pass in the future.
“CNN filed a lawsuit against the Trump Administration this morning in DC District Court,” the statement read. “It demands the return of the White House credentials of CNN’s Chief White House correspondent, Jim Acosta. The wrongful revocation of these credentials violates CNN and Acosta’s First Amendment rights of freedom of the press, and their Fifth Amendment rights to due process. We have asked this court for an immediate restraining order requiring the pass be returned to Jim, and will seek permanent relief as part of this process.”
CNN also asserted that other news organizations could have been targeted by the Trump administration this way, and could be in the future.
“While the suit is specific to CNN and Acosta, this could have happened to anyone,” the network said. “If left unchallenged, the actions of the White House would create a dangerous chilling effect for any journalist who covers our elected officials.”
Acosta has continued to do part of his job, contacting sources and filing stories, but he has been unable to attend White House events or ask questions in person — a basic part of any White House correspondent’s role.
Acosta is on a previously scheduled vacation this week. He declined to comment on the lawsuit.
On CNN’s side, CNN Worldwide chief counsel David Vigilante is joined by two prominent attorneys, Ted Boutrous and Theodore Olson. Both men are partners at Gibson, Dunn & Crutcher.
Last week, before he was retained by CNN, Boutrous tweeted that the action against Acosta “clearly violates the First Amendment.” He cited the Sherrill case.
“This sort of angry, irrational, false, arbitrary, capricious content-based discrimination regarding a White House press credential against a journalist quite clearly violates the First Amendment,” he wrote.
David McCraw, the top newsroom lawyer at The New York Times, said instances of news organizations suing a president are extremely rare.
Past examples are The New York Times v. U.S., the famous Supreme Court case involving the Pentagon Papers in 1971; and CNN’s 1981 case against the White House and the broadcast networks, when CNN sued to be included in the White House press pool.
The backdrop to this new suit, of course, is Trump’s antipathy for CNN and other news outlets. He regularly derides reporters from CNN and the network as a whole.
Abrams posited on “Reliable Sources” on Sunday that CNN might be reluctant to sue because the president already likes to portray the network as his enemy. Now there will be a legal case titled CNN Inc. versus President Trump.
But, Abrams said, “this is going to happen again,” meaning other reporters may be banned too.
“Whether it’s CNN suing or the next company suing, someone’s going to have to bring a lawsuit,” he said, “and whoever does is going to win unless there’s some sort of reason.”
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