Biogen Completes Rolling Submission of New Drug Application to FDA

I AM HAPPY TODAY FROM THIS NEWS!

Biogen and Ionis today announced that Biogen has completed the rolling submission of a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for the approval of nusinersen, an investigational treatment for spinal muscular atrophy (SMA). Biogen has also applied for Priority Review which, if granted, would shorten the review period of nusinersen following the Agency’s acceptance of the NDA.

“Since announcing the positive results of the ENDEAR interim analysis in infantile-onset SMA last month, we have heard from many families expressing their excitement about nusinersen. Their stories continue to inspire us and they are in the forefront of our minds as we work to support the FDA’s review of nusinersen,” noted Alfred Sandrock, M.D., Ph.D., executive vice president and chief medical officer at Biogen. “We appreciate the FDA’s collaboration with us during the application process, and we look forward to continuing this productive dialogue, with the goal of rapidly bringing the first treatment for SMA to as many patients as possible.”

In addition to the NDA filing with FDA, Biogen plans to submit a Marketing Authorization Application (MAA) for nusinersen to the European Medicines Agency (EMA) in the coming weeks. The EMA’s Committee for Medicinal Products for Human Use (CHMP) recently granted Accelerated Assessment to nusinersen, which can reduce the standard review time. Biogen will initiate regulatory filings in other countries in the coming months.

NEXT:http://www.curesma.org/news/biogen-completes-nda-submission.html

The Chan Zuckerberg Initiative: Together we can cure all disease by the end of the century.

Priscilla and I just shared our next major focus for the Chan Zuckerberg Initiative.

Can we help scientists to cure, prevent or manage all diseases within our children's lifetime?

I'm optimistic we can. And I hope you'll join us on this journey to a better future.

Medicine has only been a modern science for about a century, and we've made incredible progress so far. Life expectancy has increased by 1/4 of a year per year since then, and if we only continue this trend, the average will reach 100 around the end of this century.

Today, just four kinds of diseases cause the majority of deaths. We can make progress on all of them with the right technology.

Throughout history, most scientific breakthroughs have been preceded by the invention of new tools to help us see problems in new ways -- like the telescope, the microscope and DNA sequencing.

It's not hard to imagine the modern tools required to accelerate breakthroughs in today's four major disease area. So we're going to focus on bringing scientists and engineers together to build these new tools and technologies.

Today, we announced a few steps in this direction:

Dr. Cori Bargmann, a world-renowned expert in neuroscience and genetics, is joining the Chan Zuckerberg Initiative to lead this initiative. We are thrilled to welcome her.

We are committing to invest $3 billion over the next decade in this initiative to help scientists cure diseases.

Our first project is creating the Biohub. We're investing $600 million in a new research hub to bring scientists and engineers together from Stanford, UCSF, Berkeley, and the world-class engineering team we're building at the Chan Zuckerberg Initiative, in order to build some of the new tools I mentioned above.

The science initiative is a long term effort. We plan to invest billions of dollars over decades. But it will take years for these tools to be developed and longer to put them into full use. This is hard and we need to be patient, but it's important.

This is about the future we want for our daughter and children everywhere. If there's a chance that we can help cure all diseases in our children's lifetimes, then we will do our part. Together, we have a real shot at leaving the world a better place for our children than we found it.

https://actionsprout.io/404D13/thanks

International Day of Peace

Today is the International Day of Peace. World Day of Love.

I love everyone with SMA!

Trial of SMA Therapy RG7800 on Hold, but Roche, Partners Developing Another Compound September 20, 2016 Charles Mooreby Charles Moore In News.

International pharmaceutical company Roche, with its SMA drug development collaboration partners PTC Therapeutics and the SMA Foundation, said a clinical study called Moonfish (NCT02240355) that was investigating a compound and investigational medicine known as RG7800 for people with spinal muscular atrophy (SMA) was placed on clinical hold in April 2015 after an unexpected eye finding in an animal study

Roche, PTC, and the SMA Foundation announced the hold in an update on the clinical development program of SMN2 splicing modifier drug development program for treating SMA.

However, the partners are developing another SMN2 splicing modifier compound called RG7916 that is still in early clinical stages.

Roche said that concentrations of RG7800 being tested in the animal study were higher than those received by any of the Moonfish study’s human participants, and the company says it can can confirm that no safety issues have been identified in any person who received RG7800 through their participation in the Moonfish study.

RG7800 is an investigational oral drug for the treatment of SMA, a genetic disorder caused by the mutation or deletion of the survival of motor neuron gene 1 (SMN1).

curesmalogoRoche and its partners believe RG7800 may have potential to target SMA’s underlying cause by increasing SMN protein levels in the nervous system, muscles, and other tissues through modifying SMN2 gene splicing to stimulate greater production of full-length SMN mRNA.

The SMN2 splicing modifier drug development program was originally launched by PTC Therapeutics in partnership with the SMA Foundation. The program was designed to utilize PTC’s Alternative Splicing technology platform to identify and develop new small-molecule therapeutics in treating and prevention of SMA.

In November 2011, Roche gained an exclusive license to the program, with clinical development of RG7800 being led by Roche with oversight provided by a steering committee with members representing Roche, PTC, and the SMA Foundation.

However, with further clinical development of RG7800 still on hold, in January 2016 the collaborators began a Phase 1 clinical trial in the Netherlands of another SMN2 splicing modifier compound called RG7916.

Based on information from that study, the partners hope to begin clinical trials of RG7916 in people with SMA Type 1 and SMA Type 2/3 by the end of this year. Clinical trial sites, enrollment information, and study timelines will be announced once regulatory feedback has been obtained in countries where the proposed trials will be conducted.

Moonfish study participants will be offered the opportunity to enroll in an open label study with RG7916 provided they meet study criteria. These patients will be contacted by their clinical trial administrator to update them on the new developments.

For more information about updates on the RG7800 and RG7916 studies, visit https://www.clinicaltrialsregister.eu or https://clinicaltrials.gov

or contact the , Information Support Line at 888-662-6728.

The partners say details of the upcoming RG7916 studies will be posted on these websites soon. They note that public participation in clinical trials is critical to the process of advancing scientific understanding of SMA.

According to the research and support organization CureSMA, the disease affects approximately 1 in 10,000 babies, and is the No. 1 genetic cause of death in infants, with about 1 in every 50 Americans a genetic carrier. There is currently no cure for SMA, with treatment consisting of managing the symptoms and preventing complications.

From:http://smanewstoday.com/2016/09/20/sma-drug-rg7800-clinical-trial-on-hold-partners-developing-rg7916

5 Experimental SMA Therapies You Might Find Interesting

1. AVXS 101

Recently, AveXis’ gene therapy candidate AVXS-101, the only gene therapy for spinal muscular atrophy currently in human testing, has been granted FDA orphan drug status for treatment of all types of SMA. It was also granted FDA breakthrough therapy designation and was fast track designation.


2. CK-2127107 (CK-107)

In collaboration with Astellas, Cytokinetics is developing CK-2127107 (CK-107), a new skeletal muscle troponin activator as a potential treatment for people living with SMA. CK-107 has completed five Phase 1 clinical trials in healthy volunteers, and is currently the subject of two Phase 2 clinical trials.


3. Nusinersen

Biogen and Ionis Pharmaceuticals recently announced that nusinersen, their investigational drug for treating SMA, met the primary end point for the interim analysis of the ENDEAR Phase 3 trial evaluating nusinersen in infantile-onset SMA.


4. RG7800

PTC’s more developed SMA drug candidate, RG7800, is the subject of a Phase 2 trial in adult and pediatric SMA patients, but dosing was suspended and the trial was placed on clinical hold to investigate a non-clinical safety finding observed in a longer-term animal study.


5. Gene Therapy

Learn more about how gene therapy can be used to treat SMA:http://smanewstoday.com/tag/gene-therapy/

http://smanewstoday.com/social-clips/2016/09/06/5-sma-experimental-therapies-you-should-know-about/5/

Promising New Drug Could Help Treat Spinal Muscular Atrophy

According to studies, approximately one out of every 40 individuals in the United States is a carrier of the gene responsible for spinal muscular atrophy (SMA), a neurodegenerative disease that causes muscles to weaken over time. Researchers at the University of Missouri developed a new molecule in April 2014 that was found to be highly effective in animal models exhibiting SMA. Now, testing of that compound is leading to a better prognosis for mice with the disease and the possibility of potential drugs that will improve outcomes for patients with SMA.

"Our team has been fine-tuning a potential therapeutic for SMA and what it does," said Chris Lorson, an investigator in the Bond Life Sciences Center and a professor of veterinary pathobiology in the MU College of Veterinary Medicine. "It's a type of molecule called an antisense oligonucleotide, or ASO, that essentially is synthetic string of nucleic acid that binds a specific sequence in the gene."

In individuals affected by SMA, the survival motor neuron-1 (SMN1) gene is mutated and lacks the ability to process a key protein that helps neurons function. Muscles that control walking or even lifting an arm often are profoundly affected as well as muscles important for breathing. Fortunately, humans have a nearly identical copy gene called SMN2, however, SMN2 normally only makes a small amount of the correct SMN protein. Lorson's compound targets SMN2 and effectively "turns the volume up" for SMN2, allowing it to make more of the correct SMN protein.

"Our current treatment helps the body create a backup mechanism to combat the disease and extends survival in mice with SMA from just 13 days to a little over five months after only one injection at birth," Lorson said. "This treatment helps produce the right form of SMN, the one that was only produced at very low levels before."

Lorson stressed that his lab's achievement does not promise a cure for SMA and that it is unlikely a single compound will address the full gamut of symptoms. However, by combining therapies currently being researched, a better prognosis could be on the horizon, Lorson said.

The early-stage results of this research are promising. If additional studies are successful within the next few years, these compounds may be tested in human clinical trials with the hope of developing new treatments for SMA.

The study, "Optimization of Morpholino Antisense Oligonucleotides Targeting the Intronic Repressor Element1 in Spinal Muscular Atrophy," recently was accepted for publication inMolecular Therapy, a journal of Nature. Previous funding was received from CureSMA. Erkan Osman, a postdoctoral fellow and lead author on this publication working in Lorson's lab is funded by FightSMA and the Gwendolyn Strong Foundation. The content is solely the responsibility of the authors and does not necessarily represent the official views of the funding agencies.

http://www.dddmag.com/news/2016/07/promising-new-drug-could-help-treat-spinal-muscular-atrophy#.V9NWc1zhXmc.facebook

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