New MS drug approval delayed by three months

orange

People with MS who have been looking forward to the long-awaited US Food and Drug Administration (FDA) approval of the MS drug Ocrevus (ocrelizumab) are disappointed now it has been delayed. This is because, although a decision had been promised for late December 2016, an 11th-hour decision means the approval hearing has now been put back to late March 2017. I can understand that disappointment.

Manufacturer Genentech, said: “The extension is the result of the submission of additional data by Genentech regarding the commercial manufacturing process of Ocrevus, which required additional time for FDA review. The extension is not related to the efficacy or safety of Ocrevus.”ocrelizumab

Across the Atlantic, ocrelizumab is currently being reviewed for licensing in Europe as a treatment for both primary progressive and relapsing MS. Its long-term safety profile will need to be investigated, in further trials and in the real world. A decision by the European Medicines Agency (EMA) is expected in late 2017.

The full results of the latest ocrelizumab US trials have been published in the New England Journal of Medicine. These results have previously been presented at conferences, but this is the first time they have been published in a peer-reviewed journal, which is the gold standard in academic research.

Both the UK’s MS Society and MS Trust welcomed publication of the trial data prior to the FDA delay being announced.

MS Society head of clinical trials Dr Aisling McMahon said: “This (trial data) is really big news for people with the primary progressive form of multiple sclerosis. It’s the first time a treatment has shown the potential to reduce disability progression for this type of MS, which offers a lot of hope for the future.

“MS can be challenging and unpredictable and the 15,000 people in the UK living with primary progressive MS currently have no treatments available to slow the worsening of their condition. Before this treatment is available on the NHS it needs to be licensed by the European Medicines Agency and assessed for cost-effectiveness.”

MS Trust chief executive Pam Macfarlane said: “This is very encouraging news for people with PPMS. There has never been a positive result in a phase III trial of this kind and it is cause for greater hope that there is a treatment that could have an effect on disability.  We look forward to hearing more detail about the trial and whether the positive effects on disability can be sustained over the longer term.”

If ocrelizumab is licensed in Europe, the UK’s National Institute and Care Excellence (NICE) and the Scottish Medicines Consortium (SMC) will then decide whether to make it available on the National Health Service (NHS).

This decision will be based on how cost effective the treatment is. NICE will look at both the price and how much it can help people. Ocrelizumab is also being considered for licensing by the US Food and Drug Administration in the USA.

• Ocrelizumab is being developed for both relapsing and primary progressive MS. This research is notable for being the first large scale study to report a reduction in disability progression in primary progressive MS. 160 of the 487 people in the ocrelizumab group had confirmed progression at 12 weeks, compared to 96 of the 244 people in the placebo group. In relapsing remitting MS, ocrelizumab reduced the number of relapses by 50 percent compared to interferon beta 1a (Rebif).

strap-new

ian-skype_edited50shadesofsun.com is the personal website of Ian Franks, who is Managing Editor (columns division) of BioNews Services. BioNews is owner of 50 disease/disorder-specific news and information websites – including MS News Today. Ian has enjoyed a successful career as a journalist, from reporter to editor, in the print media. During that career he gained a Journalist of the Year award in his native UK. He was diagnosed with MS in 2002 but continued working until mobility problems forced him to retire early in late 2006. He now lives in the south of Spain. Besides MS, Ian is also able to write about both epilepsy and cardiovascular matters from a patient’s perspective and is a keen advocate on mobility and accessibility issues.

Homeopathy ‘treatments’ may have labels to warn they do not work

ms-header

Homeopathic ‘medicines’, including those used by some people with MS, could soon bear labels saying that they may not work – by order of the US government.

Homeopathic remedies are regulated as drugs under the Federal Food, Drug and Cosmetic Act (FDCA). Under current Agency policy, the Food and Drug Administration (FDA) does not evaluate the remedies for safety or effectiveness but is now is requiring producers to provide proof of their effectiveness.

And, if the producers don’t provide such proof, their homeopathic medicines will need to carry warnings saying there is “no scientific evidence that the product works”.

According to a report by Andrew Buncombe, in the British newspaper The Independent, a notice issued by the Federal Trade Commission explained: “Homeopathy, which dates back to the late-eighteenth century, is based on the view that disease symptoms can be treated by minute doses of substances that produce similar symptoms when provided in larger doses to healthy people.”

The report continues:

Many homeopathic products are diluted to such an extent that they no longer contain detectable levels of the initial substance. In general, homeopathic product claims are not based on modern scientific methods and are not accepted by modern medical experts, but homeopathy nevertheless has many adherents.

homeopathicSlate said there was near-unanimous mainstream scientific consensus that homeopathy’s purported mechanism of action – using ultra-highly diluted substances to allow “like to cure like” – runs counter to basic principles of chemistry, biology, and physics.

Health policy expert Timothy Caulfield recently said: “To believe homeopathy works … is to believe in magic.”

Yet, reports suggest the so-called treatments are unlikely to disappear from the shelves of pharmacists’ shops.

The FTC said that a homeopathic drug claim that is not substantiated by competent and reliable scientific evidence “might not be deceptive if the advertisement or label where it appears effectively communicates that:

  • there is no scientific evidence that the product works; and
  • the product’s claims are based only on theories of homeopathy from the 1700s that are not accepted by most modern medical experts.

new strap

ian profile50shadesofsun.com is the personal website of Ian Franks, who is Managing Editor (columns division) of BioNews Services. BioNews is owner of 50 disease/didorder-specific news and information websites – including MS News Today. Ian has enjoyed a successful career as a journalist, from reporter to editor, in the print media. During that career he gained a Journalist of the Year award in his native UK. He was diagnosed with MS in 2002 but continued working until mobility problems forced him to retire early in late 2006. He now lives in the south of Spain. Besides MS, Ian is also able to write about both epilepsy and cardiovascular matters from a patient’s perspective and is a keen advocate on mobility and accessibility issues.

Welcome or Not, FDA Focuses on Stem Cell Treatments

Drug ´breakthrough´ for primary progressive MS

roche logo

Everyone living with primary progressive multiple sclerosis should be excited by the fact a drug being developed for treatment of that particular type of MS has been given ´breakthrough´ designation by the USA´s Food and Drug Administration (FDA).

The special status means that the drug, called ocrelizumab, could be approved more quickly following positive phase three trial results.

Breakthrough Therapy Designation exists to speed up the development and review of medicines intended to treat serious or life-threatening diseases. Fast tracking the approval process helps to ensure people have access through the US drug approval pathway as soon as possible.

The timing of any final approval is not yet known but once the FDA grants ocrelizumab a licence this means it will be available for doctors to prescribe in the US. Though the UK is part of a separate licensing process in Europe this is still a significant step forward.

The ´breakthrough designation has been granted based on top line results presented by pharmaceutical company Roche.

Ocrelizumab is the very first treatment to have shown positive results in a phase three clinical trial for people with primary progressive MS.

Treatment with ocrelizumab led to a reduction in the progression of clinical disability by 24% compared to placebo. This reduction was sustained for at least 12 weeks and was measured by the Expanded Disability Status Scale (EDSS).

In relapsing remitting MS, ocrelizumab reduced the number of relapses, by 46% in one trial and 47% in another when compared to Rebif.

If GM creatures are ‘safe’, why worry about ‘negligible risk’ of escape?

gm salmon

Two stories in the news are currently giving me a few concerns. At first glance, they may appear unconnected but they are linked by the facts that they involve genetically modified creatures and that one has already been approved while a field test of the other is being supported by a parliamentary scientific committee.

Now, we have heard a great deal about genetically modified (GM) crops and the subject has been the cause of a protests, debate and international restrictions. To some, GM is a solution to the world food shortage while to others it is a step fraught with dangers, a step into the unknown, a frightening step too far.

I will not knowingly eat anything that includes a genetically modified ingredient and, when I used to have a smallholding, I always bought GM-free animal feed.

The first news story giving me concern is the decision of the USA’s FDA (Food and Drug Administration) to approve GM Atlantic Salmon as suitable for human consumption while at the same time saying that there is no need to label it as being genetically modified.

The decision only relates to two land-based breeding and production facilities, so far, in Canada and Mexico. Maybe I am being cynical, but isn’t it strange that both sites are outside the USA even though the company involved is based in Massachusetts?

Further, apparently, there is almost no chance, a negligible risk, of any GM fish escaping and mixing with natural fish. Almost no chance? That means there IS a chance, however small. And, if the GM salmon is so safe, why even be worried about any escaping?

The ‘good news’, however, is that all the GM fish will be sterile females – but more about that later!

The second story is that a scientific committee of the UK parliament’s upper chamber, the House of Lords, has called on the government to support a field trial of GM insects. It claims they could provide safer alternatives to pesticides or even eliminate transmission of lethal diseases.

Such a move has been the subject of a ban by European Union regulations but the committee feels that this ban has already been broken and that Britain should now take a lead.

But where would it go? How can a field trial be controlled? How can GM insects be prevented from mixing with natural ones? Ah, but they would all be sterile!

Lord Selborne, who chairs the committee, said that companies need to be encouraged as GM insect technology has the potential to save countless lives worldwide and to generate significant economic benefits for Britain. Potential to save lives? Maybe but also a potential for unknown disaster too.

Mark Shadlow, chief executive of conservation group Buglife, said: “We are surprised that the report didn’t summarise the environmental risks that were highlighted in its evidence gathering and instead recommended a UK ‘field trial’, without making a clear case for a UK problem that needs to be tackled or, therefore, the type of insect to be trialled.”

In both cases, though, the GM creatures are said to be sterile (female salmon and male insects) and incapable of reproduction. If they are so ‘safe’, I wonder why such precautions are even considered necessary.

Environmentalists have long opposed the plan, saying the modified fish could escape its containment and mix with wild salmon populations.

The FDA said an escape is almost impossible because of redundant measures put in place to contain them in tanks. It also says that because the fish are sterile, they would not be able to breed with wild salmon.

But is sterility any guarantee? Not according to Canadian scientists who have found that the GM salmon ‘are capable of hybridizing with wild brown trout, creating offspring that carry modified growth genes and outcompete both wild and GM fish’.

Even if successfully kept apart from other creature, is ‘all same gender’ any real safeguard against reproduction? Well, no, not really. Nature has a number of examples of creatures that can change sex to overcome a shortage of one of them.

Molly Edmonds, writing in How Stuff Works – Health, says, “Reproductive success and continued survival are the main reasons that species change sex. The change could also occur due to environmental factors or chemical triggers that scientists don’t understand yet.”

 

Pic: A genetically modified Atlantic salmon with a natural salmon of the same age. Picture by Aquabounty, the Massachusetts-based company behind the GM salmon project.

 

Note: The author of this blog was named Farming Journalist of the Year in Wales, UK, while Rural Affairs Editor of a regional newspaper group, in 1999.