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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.”

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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.

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Duchenne muscular dystrophy drugs at the crossroads, as newer agents advance

NATURE BIOTECHNOLOGY
VOLUME 34 NUMBER 7 JULY 2016 p675

The first generation of exon-skipping drugs for Duchenne muscular dystrophy (DMD) has reached a critical juncture, as an FDA decision on whether to grant accelerated approval to Sarepta Therapeutics’ controversial drug Exondys (eteplirsen) looms. A rival effort at BioMarin Pharmaceutical, of San Rafael, California, is now over. Biomarin withdrew a European application for Kyndrisa (drisapersen) on May 31, after unsuccessful talks with European Medicines Agency officials, and the US Food and Drug Administration (FDA) refused to approve the same drug in January.

The refusals were unequivocal—drisapersen was neither safe nor effective. In the case of Sarepta’s eteplirsen, however, regulatory uncertainty abounds. The Cambridge, Massachusetts–based biotech, is seeking approval with evidence from just 12 patients. “In any other therapeutic area, the agency would be within its rights to
We can counteract this effect by simply increasing supply of blood to the penis; however, levitra samples free they do not target the very reasons for erectile dysfunction. L-Arginine was considered to be “nature’s generic levitra” for both sexes. Remember it’s your sexual health which should be chosen among a wide range of the drugs? Selecting only best drug can help a patient relieving his condition cialis 10 mg http://nichestlouis.com/cialis-2733.html safer and faster. This gives patients confidence in the healthcare viagra in uk Continue to pharmacy offered by India generally as they are already used to the expertise and professionalism of Indian medical staff. say this data set is very exciting—come back when you’ve conducted a trial which proves it’s safe and effective,” says Glyn Edwards, CEO of Abingdon, UK–based Summit Therapeutics.

“The normal balance of evidence hasn’t been achieved for this drug yet.”

Click Here to see the full story

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New data underscore benefits of idebenone in DMD as Biomarin pulls drisapersen MAA

BIOWORLD TODAY

By Cormac Sheridan Staff Writer

Thursday, June 2, 2016

DUBLIN – Even as Biomarin Pharmaceutical Inc.’s dwindling hopes for its exon-skipping pipeline in Duchenne muscular dystrophy (DMD) turned to ash this week, with the withdrawal of its marketing authorization application (MAA) for drisapersen from the EMA, there was also some positive news for DMD patients and their families. Santhera Pharmaceuticals AG released additional clinical data from its Delos phase III study of Raxone (idebenone) in Duchenne muscular dystrophy (DMD), which showed that the drug’s ability to slow the rate of decline of respiratory function over one year converted into a range of clinical benefits.
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The new analysis forms part of its recently completed regulatory filing in Europe and its planned filing in the U.S. The Liestal, Switzerland-based company filed for approval in DMD in Europe Tuesday, as a type II variation to its existing approval for treating visual impairment in Leber’s hereditary optic neuropathy. If the FDA is agreeable, it could complete a rolling new drug application during the third quarter of 2016 – although that depends on the outcome of a meeting in late July.

The primary endpoint of the Delos trial, which recruited 64 DMD patients who were not on concomitant glucocorticoid therapy, was the alteration in lung function over the one-year trial, as measured by the percentage change from baseline to week 52 in peak expiratory flow (PEF) for each patient. PEF for those in the drug treatment group declined by an average 2.57 percent, whereas it declined by an average 8.84 percent for those in the control group. (See BioWorld Today, May 14, 2014.)

Click Here to read the full article

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Duchenne candidate Raxone (idebenone)

fiercebiotech

 

 
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May 4, 2016

Santhera Pharmaceuticals has announced a slew of updates in regards to regulatory filings for its Duchenne candidate Raxone (idebenone). The Swiss biotech said it plans to submit “comprehensive briefing material” and a meeting request to the FDA to discuss filing an NDA for DMD patients who are not taking concomitant glucocorticoids. Santhera said it will also submit a new marketing application for Raxone in Duchenne to the EMA. In addition, it’s reporting the launch of a new Phase III (SIDEROS) clinical trial in DMD using glucocorticoids. Patient enrollment will start in coming weeks

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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
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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

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Santhera Drug Wins European Approval for Rare Blindness Disease

By Adam Feuerstein Follow | 09/09/15 – 01:01 AM EDT

http://www.thestreet.com/story/13280711/1/santhera-drug-wins-european-approval-for-rare-blindness-disease.html

Santhera

Liestal, Switzerland (TheStreet) — European regulators approved Wednesday a new drug from Santhera Pharmaceuticals (SANN.SW) to treat a rare, inherited disease which causes progressive vision loss and blindness.

The Santhera drug, Raxone, is the first approved treatment for Leber hereditary optic neuropathy (LHON), which affects approximately 10,000 patients (mostly males) in Europe.

The Raxone commercial launch will commence in the coming weeks, said Santhera CEO Thomas Meier. He declined to discuss how much the company expects to charge for Raxone except to say that it will be “priced as a typical orphan disease drug.”
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Raxone is a pill designed to stimulate mitochondria, the energy-producing organelle found inside cells. In LHON, a defect in the mitochondrial DNA causes retinal ganglion cells — a type of nerve cell connecting the eye to the brain — to lose energy and stop functioning. When retinal ganglion cells stop working, patients experience progressive vision loss and eventual blindness.

Raxone doesn’t correct the defective mitochondrial DNA underlying LHON. Instead, the drug circumvents the genetic defect and restores energy levels to the retinal ganglion cells. European regulators approved Raxone based on data from a clinical trial and an expanded access program showing the drug mitigates and reverses vision loss in LHON patients.

RBC Capital analyst Simos Simeonidis forecasts $29 million in peak Raxone LHON sales based on annual pricing of $43,000.

The LHON indication makes up just a fraction of RBC’s Santhera valuation because the company is also developing Raxone to improve the lung function of Duchenne muscular dystrophy (DMD) patients. The progressive weakening of muscles in the chest of DMD patients leads to respiratory disease and breathing problems. Santhera hopes to submit Raxone approval filings for DMD in the U.S. and Europe by the end of the year, said Meier.

Click Here to read a PDF version.

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CHMP backs Santhera’s Raxone in hereditary blindness condition

BIOWORLD
THE LHON AND WINDING ROAD
CHMP backs Santhera’s Raxone in hereditary blindness condition

By Cormac Sheridan Staff Writer
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Monday, June 29, 2015
DUBLIN – After a long and sometimes perilous review process, Santhera Pharmaceutical Holding AG has finally gotten Raxone (idebenone) across the line. At the second time of asking, the EMA’s Committee for Human Medicinal Products (CHMP) voted in favor of its approval in Leber’s hereditary optic neuropathy (LHON), paving the way for formal European Commission approval just over two months from now.

Click here to read the full article…

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PATIENTS LEAD THE WAY

Click here to read the full article…

BY EMILY CUKIER-MEISNER, SENIOR WRITER

FDA’s draft guidance on Duchenne muscular dystrophy and related
dystrophinopathies is the culmination of an extensive e6ort led by a
patient advocacy group that enlisted the support of patients, caregivers,
academia and industry.
DMD drug development has been hindered by a lack of defined outcome
measures and understanding of surrogate markers or biomarkers that
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Muscular Dystrophy (PPMD) and the DMD community to submit a
proposed draft guidance that could clarify a clinical development pathway
for approval.
FDA spokesperson Kristofer Baumgartner said in a statement to
BioCentury that PPMD’s submission is the first time in the Center for
Drug Evaluation and Research’s knowledge that an advocacy group has
submitted a proposed draft guidance, and that this type of engagement is
an example of how early input from patients and caregivers can contribute
to drug development.

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Duchenne Muscular Dystrophy: Idebenone Slows Respiratory Decline in Phase 3 Trial

NEUROLOGY TODAY

http://journals.lww.com/neurotodayonline/blog/NeurologyTodayConferenceReportersAANAnnualMeeting/pages/post.aspx?PostID=11&cid=MR-eJP-AANConference-Day4US-Neurology-WNT-NoPromo&desktopMode=true

Friday, April 24, 2015

April 24—Duchenne Muscular Dystrophy: Idebenone Slows Respiratory Decline in Phase 3 Trial

BY RICHARD ROBINSON

WASHINGTON—Idebenone slows the decline of respiratory function in boys with Duchenne muscular dystrophy (DMD), according to a yearlong, double-blind phase 3 trial in 64 boys. Lead investigator Gunnar Buyse, MD, PhD, a professor of pediatrics and child neurology at the University of Leuven in Belgium, presented the results here on Friday at the AAN Annual Meeting.

Boys receiving 900 mg per day of idebenone for 52 weeks experienced a non-significant decline in peak expiratory flow as percent predicted (PEF%p; -3%p), the primary outcome measure, compared with a significant decline (-9%p) in those receiving placebo. This represents a 66% reduction in loss of PEF%p. PEF%p is a measure of peak flow (respiratory strength) compared with height -corrected norms for the population. Other respiratory measures were also significantly different between the two groups.

“This is the first ever successful phase 3 trial in this complex disease,” Dr. Buyse told the Neurology Today Conference Reporter before the session.

The major reason behind the problem of impotence is generic viagra soft no more an issue of concern but if the problem persists for half or more times you encounter a sexual intercourse then the problem demands treatments. Causes prompting this health cialis 100mg pills issue in men include relationship issues, depression, prostate gland injury, vascular disorder, etc. sildenafil price Ammonia itself is a by-product of intensive exercise, and without the urea cycle the body would rapidly become polluted. However, cialis from india tadalafil working through such issues with a partner can help for preventing impotence condition. Idebenone is a synthetic quinone compound with both electron transfer and antioxidant effects, Dr. Buyse said. “We know that in DMD cells there is a problem with oxidative stress, as well as with energy production.” Idebenone works in the mitochondrial electron transport chain to increase energy production and reduce oxidation, he explained.

In the mdx mouse model of DMD, idebenone has been shown in blinded experiments to improve cardiac function over nine months of treatment, as well as to increase the level of voluntary exercise. A successful phase 2 trial of idebenone in DMD set the stage for the current trial.

Enrolled patients were between 10 and 18 years old and were not currently taking steroids; about 90 percent had progressed to requiring a wheelchair for mobility. Peak expiratory flow was chosen as the primary endpoint after conferring with regulators, Dr. Buyse said. It is a direct measure of strength or weakness of the expiratory muscles, unlike forced vital capacity, which can also reflect the degree of scoliosis or other comorbidities. “There is good evidence that peak flow is an earlier and more sensitive parameter than forced vital capacity,” he said.

In addition to the effect on peak expiratory flow, the benefits of treatment were seen on forced vital capacity and forced expiratory volume in one second. The researchers observed no difference in benefit between boys who had or had not previously used steroids. Treatment was safe and well tolerated. “It is really a coherent picture showing the beneficial effect of the intervention,” Dr. Buyse said.

Based on the results from this trial, the investigators plan to apply for regulatory approval in both the United States and Europe. If the drug is approved, it will be considered for use earlier in the disease than was tested in the trial. “In principle, there is no reason idebenone would not work earlier,” Dr. Buyse said. Based on both its mechanism of action and some subanalyses showing that younger patients in the trial benefited equally compared with older ones, he added, “there is every reason to think that the sooner you start it, the better it would work.”

Kathryn R. Wagner, MD, PhD, director of the Center for Genetic Muscle Disorders at the Kennedy Krieger Institute in Baltimore, MD, cautioned, however, that these results apply directly only to boys not currently taking steroids. “From this trial, we do not know if idebenone will provide benefit to boys who are receiving this standard-of-care treatment,” said Dr. Wagner, who was not involved with the study.

A further caveat is that PEF%p “is not a measure that is used clinically in DMD, and it is difficult to know its relevance,” Dr. Wagner said, adding that this could be a concern unless it can be shown to be predictive of a more clinically relevant measure, such as the need for ventilation or decreased respiratory infections.

Nonetheless, she said, “Novel drugs are desperately needed in Duchenne muscular dystrophy. We assume that drugs that improve skeletal muscle function will improve pulmonary function, but this may be an oversimplification. A trial focusing on adolescents and pulmonary function is therefore welcomed.”

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Results from Phase III trial of Raxone/Catena in patients with DMD published

euroPharmReview

21 April 2015  •  Author: Victoria White

Home

Santhera Pharmaceuticals have announced that the full results of the double-blind placebo-controlled Phase III trial (DELOS) demonstrating efficacy and safety of Raxone®/Catena® in patients with Duchenne muscular dystrophy (DMD) have been published in The Lancet.

euroPharmReview2The results of the DELOS trial demonstrated that Raxone/Catena (INN: idebenone) significantly reduced the annual decline in Peak Expiratory Flow (PEF as percent predicted, PEF%p) by 66% compared to patients taking placebo. Other respiratory function endpoints such as Forced Vital Capacity (FVC) and Forced Expiratory Volume (FEV1) corroborated these results and showed a consistent pattern with treatment differences supporting efficacy of Raxone/Catena over placebo in the preservation of respiratory function. Researchers concluded that Raxone/Catena represents a new treatment option for DMD patients.

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Gunnar M. Buyse, MD, PhD, Professor of Child Neurology at the University Hospitals Leuven (Belgium) and Principal Investigator for the DELOS trial, said, “Statistically significant and clinically relevant outcomes of primary and secondary endpoints coherently demonstrated that Raxone/Catena reduced the loss of respiratory function and that it was safe and well tolerated. I am very enthusiastic about the positive data from the trial which demonstrate that this drug represents a suitable treatment option to ameliorate a life-threatening complication of the disease.”

“With morbidity and mortality in DMD being associated with progressive restrictive lung disease and irreversible loss of lung function, these findings represent an important treatment effect and are of major clinical relevance for patients with DMD”, added Craig McDonald, MD, Professor and Chair of the Department of Physical Medicine & Rehabilitation at UC Davis (USA) and investigator of the DELOS trial.

“The degree of slowing of respiratory function loss demonstrated in DELOS is of major clinical relevance for patients with DMD”, commented Nicholas Coppard, PhD, SVP Development at Santhera. “Based on this benefit and its well-established safety profile, we are very excited about the prospects of Raxone/Catena as a treatment option for DMD patients and we are currently preparing the regulatory filing dossier for application of marketing authorization both in the US and Europe.”