Posted on

Study of Potential Therapy for Congenital Muscular Dystrophy Gets Boost from FDA

msnewstodayStudy of Potential Therapy for Congenital Muscular Dystrophy Gets Boost from FDA

August 31, 2016

By Carolina Henriques

http://musculardystrophynews.com/2016/08/31/fda-grant-supports-clinical-trial-of-congenital-muscular-dystrophy-treatment-omigapil/

Santhera Pharmaceuticals announced that it has received a $246,000 grant from the U.S. Food and Drug Administration (FDA) to support its ongoing Phase 1 clinical trial evaluating omigapil as a potential treatment for children and adolescents with congenital muscular dystrophy (CMD).

The study, called CALLISTO (NCT01805024), is assessing the pharmacokinetics, safety, and tolerability of omigapil in ambulatory and non-ambulatory children, ages 5–16, with either of two CMD subtypes (COL6-RD or LAMA2-RD). About 20 eligible patients are currently being recruited; more information is available on the study’s clinical trials.gov website.

“We are delighted that the FDA has awarded us this prestigious and highly competitive grant in support of our CALLISTO trial in CMD,” Thomas Meier, Ph.D., chief executive officer of Santhera, said in a press release. “With this award the FDA emphasizes the need for a therapy for CMD and the contribution of the CALLISTO trial to the development of an effective treatment.”

The symptoms of stress may be divided in to broad categories: cognitive, physical, emotional and behavioral. buy viagra without rx You can’t afford to continue suffering silently while your relationships are going cialis for women through misunderstandings, then it is better to clear them and cuddle each other. find out my website buy levitra It can get a man engrossed well in sexual intercourse, making for steamy time with partner. Sildenafil Citrate is responsible making the blood flow to the penile region after the viagra generico uk stimulation. Severe forms of CMD can cause life-threatening progressive muscle weakness, known as floppy infant syndrome. A contributing factor for disease progression is apoptosis, or cell death, through a cascade of cellular events involving the glyceraldehyde-3-phosphate dehydrogenase (GAPDH) pathways for the transcription of pro-apoptotic genes.

CMD is a clinically distinct group of inherited neuromuscular diseases that manifests with an early onset of symptoms that include weakness, contractures, elevated creatine kinase levels, an inability to stand or walk, eating difficulties, and respiratory insufficiency.

Omigapil is a deprenyl-analog with anti-apoptotic properties that binds to GAPDH. This interaction leads to its inhibition, and is thought to underlie the compound’s observed anti-apoptotic effect. In animal models of CMD, omigapil was seen to prevent cell death, reduce body weight loss and skeletal deformation, and to improve locomotive activity.

The proposed treatment was given Fast Track designation by the FDA for the treatment of CMD in May, and has previously been granted Orphan Drug Designation for the same indication in Europe and the United States. Santhera’s grant award came from the FEA’s Office of Orphan Products Development.

“As the leading organization representing the interests of patients with CMD, we are very pleased that the FDA recognizes the need to advance medical research in this orphan disease. With currently no effective treatment available for patients, the CALLISTO trial offers hope to patients with this devastating disease,” said Patrick May, CureCMD’s president.

The trial is being conducted in collaboration with the U.S. National Institutes of Health (NIH) under the leadership of Professor Carsten Bönnemann and Dr. Reghan Foley, with the National Institute of Neurological Disorders and Stroke (NINDS). Study completion is expected in early 2017.

Omigapil was developed by Novartis, and Santhera holds an exclusive worldwide license for its development as a treatment for congenital muscular dystrophy.

Posted on

Santhera holds talks with potential Eastern European distribution partners for Raxone in LHON – CEO

Click Here to See The Original Article

Reimbursement discussions ongoing in multiple countries

DMD regulatory filings expected before YE15

Santhera Pharmaceuticals (SWX:SANN) is in ongoing discussions with potential distributors in Eastern Europe for Raxone (idebenone) in Leber’s hereditary optic neuropathy (LHON), said CEO Thomas Meier. There is a reasonable chance the company will have selected a partner by the end of this year, he said.

The company received EMA marketing authorisation for Raxone in LHON on 9 September, according to a company press release.

The company has built an infrastructure for Western European countries; however, in Eastern European territories it needs logistical help, Meier said.

In terms of deal structure it is likely Santhera would pay a fee to the distribution partner and it may receive a percentage of the sales within those territories, Meier said, adding the exact arrangement is still under evaluation. However, Santhera will retain the license for Raxone in these regions, he noted. The company’s preferred scenario is to find one partner for the entire bloc of Eastern European countries rather than multiple partners for several territories, he explained.

Santhera is hiring a team of in­house experts to help commercialise Raxone within Western European territories and is hoping to launch the product in an undisclosed European country shortly, Meier said. Payer discussions are ongoing in multiple countries, he added. When asked about potential pricing, Meier declined to provide a figure however pointed to a recent analyst report which may provide an estimate. The analyst report noted an estimated net price of USD 7,373 per treatment month in 2016 and USD 7,005 for 2017 and beyond.

LHON is a heritable genetic disease causing blindness. The disease typically presents in young, otherwise healthy adults, mostly men, as rapid, painless loss of central vision in one eye, followed by visual loss in the other eye within a few months of the onset of symptoms, leading to blindness, according to a company press release.

The company will file for FDA approval for Raxone in LHON at a later date.

Additional indications
Before you start using this drug, it is imperative to do as what commander levitra the chiropractor says. Make sure you bear with us if we don’t often fit the http://foea.org/contribute/donate/ levitra viagra cialis standard “G….and G….” stereotype. Moreover, if I hear the Badfinger song “Come And Get It” (in the advertisement for discount viagra canada http://foea.org/greatness-lives-here/ a product or a service. This unfortunate result appears due to inconsistent or insufficient blood movement towards the direction of reproduction because of lessened action of cGMP enzyme. cialis no prescription
The company’s cash position at the end of August was CHF 37m, which will cover the full European launch of Raxone, Meier said. Additional financing may be required to continue development of Raxone in other indications, he noted, declining to comment on further financing details.

The company may consider similar LHON distribution agreements for Duchenne Muscular Dystrophy (DMD) in specific territories necessary, he said.

Santhera is preparing regulatory filings for Raxone in the EU and the US for DMD before the year’s end, Meier noted. On the back of the EMA approval in LHON, the company believes it may be able to apply for a line extension for Raxone in DMD which is under discussion internally. The company will initiate discussions with the Committee for Medicinal Products for Human Use (CHMP) shortly. According to the aforementioned analyst report, the LHON approval may allow for a six­month review of Raxone in DMD within Europe. The company anticipates filing its NDA to the FDA as well.

Meier declined to comment on any speculation with regards to a sale of the company and noted the management’s current preference is to stand alone and build value through obtaining additional approvals and pipeline development. Santhera is also considering additional indications for Raxone and is in discussions internally and with key opinion leaders as it believes that the drug may be beneficial for additional mitochondrial diseases, Meier said. However, he declined to name any specific indications under consideration. Within the next 12 months the company will have a clearly defined strategy for other indications, he added.

The company will not out­license Raxone in either LHON or DMD and will market alone in both the US and EU, Meier said. However, Santhera has set aside certain capital resources for in­licensing activities.

Raxone was the first approval for a mitochondrial disease and the company will consider licensing additional molecules for mitochondrial disease, neuromuscular diseases or paediatric diseases, he noted. The company could take part in codevelopment arrangements or license from a US company looking for a European partner.

Santhera also has a fully recruited Phase II trial ongoing in primary progressive multiple sclerosis, he added.

Santhera’s market cap is CHF 697m.

by Hamish McDougall in London

Email the journalist team at editorialfeedback@biopharminsight.com