AveXis Gene Therapy AVXS-101 Granted Access into EMA PRIME Program for Spinal Muscular Atrophy Type 1!!

Great news from Avexis!

CHICAGO, Jan. 31, 2017 (GLOBE NEWSWIRE) -- AveXis, Inc. (NASDAQ:AVXS), a clinical-stage gene therapy company developing treatments for patients suffering from rare and life-threatening neurological genetic diseases, today announced that the European Medicines Agency (EMA) has granted access into its PRIority MEdicines (PRIME) program for the company’s proprietary gene therapy, AVXS-101, for the treatment of spinal muscular atrophy (SMA) Type 1. The PRIME application was based on data from both preclinical evaluations and the ongoing Phase 1 clinical trial of AVXS-101 as of September 15, 2016.

PRIME is intended to enhance support for the development of medicines – specifically those that may offer a major therapeutic advantage over existing treatments or benefit patients without treatment options – through early and proactive support by EMA to optimize the generation of robust data and development plans, and potentially expedite the assessment of the Marketing Authorization Application (MAA) so these medicines may reach patients sooner.

“The acceptance of AVXS-101 into the PRIME program reflects the urgent need for innovative treatment options for the patients diagnosed with SMA in the European Union,” said James L’Italien, Ph.D., Senior Vice President, Chief Regulatory and Quality Officer of AveXis. “We are eager for this enhanced collaboration with the EMA to ensure we are taking the most appropriate and expeditious path toward the development of a robust Marketing Authorization Application submission, and to potentially streamlining the time needed to bring AVXS-101 to patients in the EU suffering from this devastating disease.”

In notifying the company of the acceptance, EMA noted: “The preliminary clinical observations following AVXS-101 administration include positive impact on survival, pulmonary function, nutritional support, preservation of motor function and the attainment of development milestones, all of which are unexpected within the framework of the natural history and disease progression for SMA Type 1. These clinically meaningful responses in the patients treated with AVXS-101 are sufficient preliminary clinical evidence of treatment effect that has the potential to address an unmet need in this devastating pediatric disease.”

In July 2016, the U.S. Food and Drug Administration (FDA) granted Breakthrough Therapy Designation, a comparable program to PRIME used by the FDA, for AVXS-101 for the treatment of patients with SMA Type 1.

AveXis intends to initiate a pivotal trial of AVXS-101 in patients with SMA Type 1 in the European Union before the end of 2017.

About PRIME

The EMA launched the PRIME initiative in March 2016 to foster research and development of medicines that may offer a major therapeutic advantage over existing treatments, or benefit patients without treatment options. PRIME aims to strengthen clinical trial designs to facilitate the generation of high quality data for the evaluation of an application for marketing authorization. To be accepted for PRIME, a medicine has to show its potential to benefit patients with unmet medical needs based on preclinical and/or early clinical data. These medicines are considered priority medicines within the European Union.

After an investigational candidate has been selected for PRIME, developers are assigned a rapporteur from the Committee for Medicinal Products for Human Use (CHMP) to provide continuous support and help to build knowledge ahead of a Marketing Authorization Application (MAA). A multidisciplinary group of experts will provide broader guidance on the overall development plan and regulatory strategy of the product. Companies are also eligible for accelerated assessment at the time of their regulatory application.

For more information, please visit the research and development section of www.ema.europa.eu

Text from: http://investors.avexis.com/phoenix.zhtml?c=254285&p=irol-newsArticle&ID=2240880

My Birthday

Yesterday I had my Birthday! I received many congratulations from many my friends from all country over the world. I am very happy.

And I continue to write in my blog about my life with SMA...

My lifestyle with SMA.

My lifestyle with SMA.

Do not forget about me. For your donation has charity accounts: PayPal: Lemeshonok@gmail.com
VISA: 4246 4100 5470 2615

Go to my group: https://www.facebook.com/groups/1183688658388473/

News from my treatment in Bologna

Hi, friends!

A few days ago I was happy event, doctor-neurologist M.Villanova from Bologna gave me breathing apparatus non-invasive ventilation. It was gift of Dr. Villanova for New Year holidays. I improve my breath for several days. I am very happy.

This photo article about my trip to Bologna.

In the spring or summer, I must again go for treatment in Bologna, now doctor decides when it will be.

So I ask you to help me find a donation for my trip for medical treatment in clinic Nigrisoli to Bologna.


My charitable accounts: PayPal:Lemeshonok@gmail.com
VISA: 4246 4100 5470 2615

Text from my page: https://www.facebook.com/groups/1183688658388473/

It was wandering event!

One year ago I have my big personal art exhibition ''Things like people''.

It was wandering event. This is day in my remember... https://www.facebook.com/groups/1183688658388473/

Your donations can save my live

Long time I don't wrote here about my treatment in Italy. Because I have problem with my health few time, I was sick 10 days and also I have new problem - I don't get needed charity donations for my trip in Italy clinic in Bologna.

Lately I have told with neurologist from Bologna and he is said that me need will to visit to clinic in April or May for urgently respiratory treatment. But I haven't financial means for it. It's very sad. I don't want refuse from this treatment, because it's very need for me.

Help me please! Your donations can save my live.

My charity account:

PayPal: Lemeshonok@gmail.com

VISA: 4246 4100 5470 2615

My charity page: https://www.gofundme.com/pleasehelpMONIKA

My charity group: https://www.facebook.com/groups/1183688658388473/

It is a shock from price for the Spinraza!

Biogen's $375K Spinraza price puts a Sovaldi-style spotlight on rare disease meds

It was big news for Biogen when its spinal muscular atrophy drug Spinraza (nusinersen) won approval just before Christmas. But in the first big drug-price controversy of the new year, the headlines really got going when Biogen unveiled Spinraza’s sticker.

The company set Spinraza’s price at $125,000 per injection, which adds up to $750,000 for the first year of treatment and $375,000 after that. Twitter lit up with critical comments—and messages urging action from lawmakers, including President-elect Donald Trump. Biogen and its development partner Ionis saw their shares drop.

Spinraza’s price isn’t far out of the ballpark in the ultra-orphan drug world, where some treatments top $400,000 per year on a list-price basis. But unfortunately for Biogen, Spinraza has made its debut at a time when public attention on drug prices is at an all-time high.

Unfortunately for other drugmakers that rely on costly rare disease meds, Spinraza’s price could bring some unwelcome attention to the entire field, just as Gilead Sciences’ $84,000 sticker price on its groundbreaking hepatitis C drug Sovaldi did for hep C and beyond. Unfortunately for patients, analysts said, the pricing decision and ensuing backlash is likely to tighten up access to the treatment.

“The sticker-shock presented in the media could turn Spinraza into the Sovaldi of rare disease drugs,” Leerink Partners analyst Geoffrey Porges wrote to investors over the weekend. It could be “the straw that breaks the camel’s back in terms of the U.S. market’s tolerance for rare disease drug pricing.”

As Piper Jaffray analyst Joshua Schimmer pointed out in a Tuesday note, the $375,000 annual cost fits within the realm of ultra-orphan disease therapies such as Alexion’s Soliris, with a list price north of $500,000 per year. In that context, “we think this is quite reasonable, especially considering the high unmet need and meaningful benefit from Spinraza,” Schimmer wrote.

That’s Biogen’s contention as well. The company “carefully considered” the price ahead of launch, spokesman Matt Fearer told CBS News. In the context of its clinical value—and Biogen’s need to fund other R&D—Spinraza’s price “is fairly in line with other therapies for rare orphan diseases,” Fearer said.

Ultra-orphan drugs tend to face much less pricing pressure than their less-expensive counterparts in other fields. That’s because the diseases they treat are so rare, only a few patients need them. The hit to payers’ budgets is relatively small, even compared with much cheaper meds used to treat thousands.

Regardless of how fair or reasonable Spinraza’s sticker might be in the ultra-orphan context, however, the outsize price tag was guaranteed to raise eyebrows, given the close scrutiny drug prices currently face. Most of Twitter’s audience—and most of the general public—doesn’t understand the economics of rare disease drugs.

Therein lies the risk, not just for Biogen and Spinraza, but other rare disease drugs and their makers. Ahead of Spinraza’s approval, Piper Jaffray included a Trump pricing tweet on its list of potential provocative events of the year. “Will Trump feel some sticker shock and rush to his Twitter account?” the analysts wrote Dec. 20. And that’s just what many on Twitter demanded over the weekend. Others tweeted to former presidential candidates Sen. Bernie Sanders and Sen. Marco Rubio, as well as other U.S. lawmakers.

At the very least, the quick pushback on Biogen’s pricing decision is likely to inspire payers to look more closely at Spinraza than they might have otherwise, Porges said. One feature of Spinraza—its standard dosing regardless of body weight—could end up a liability. Many other rare disease doses increase as weight does. And as dosing rises, so does cost, sometimes far beyond the numbers generally reported.

“This inherent price inflation dynamic is one of the ‘undiscussables’ of rare disease that drive pricing well above even the stratospheric ranges that are commonly discussed, frequently debated and occasionally justified for rare diseases,” Porges notes.

Biogen can’t benefit from those dosing-related sales increases, so the company decided to capture the value of its drug in the first-year loading phase, he said. Long term, the cost for Spinraza will be equivalent to, or even less than, that for many other rare disease drugs, but Biogen “will be unable to hide the true price in the back-end through weight-driven price escalation.”

“In turn, patient access is likely to be harmed by this price and its counter-reaction,” he said, “but the decision is ultimately not too out-of-line with other rare disease treatments with life saving, or life extending, potential.”

Information from: http://www.fiercepharma.com/pharma/biogen-s-375k-spinraza-price-puts-a-sovaldi-style-spotlight-rare-disease-meds