Spark Therapeutics Submits Marketing Authorization Application to European Medicines Agency for Investigational LUXTURNA(TM) (voretigene neparvovec)
Potential for first gene therapy for a genetic disease to be approved in both the U.S. and EU
PHILADELPHIA, July 31, 2017 (GLOBE NEWSWIRE) -- Spark Therapeutics (NASDAQ:ONCE), a fully integrated gene therapy company dedicated to challenging the inevitability of genetic disease, announced today that it has submitted a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for LUXTURNA(TM), the proposed trade name for voretigene neparvovec, an investigational, one-time gene therapy for the treatment of patients with vision loss due to Leber congenital amaurosis or retinitis pigmentosa caused by confirmed biallelic RPE65 mutations. The MAA includes data from three clinical trials that enrolled 41 participants with RPE65 -mediated inherited retinal dystrophy (IRD), including the first randomized, controlled Phase 3 trial for a gene therapy for a genetic disease.
Once EMA has validated the application, the review period will begin. Spark Therapeutics has previously received orphan product designations for LUXTURNA from EMA for the treatment of both Leber congenital amaurosis and retinitis pigmentosa.
"Today's announcement represents an important moment in the effort to treat blindness caused by inherited retinal degenerative diseases," said Christina Fasser, president of Retina International, an umbrella organization of more than 43 patient organizations world-wide promoting research in view to find a cure to inherited retinal degenerative diseases. "We are excited about the potential of this application to bring the first gene therapy to patients with this form of IRD."
The investigational therapy is under Priority Review with the U.S. Food and Drug Administration, with an assigned Prescription Drug User Fee Act (PDUFA) date of Jan. 12, 2018.
"With LUXTURNA now in regulatory review on both sides of the Atlantic, we are building out our medical and commercial infrastructure to prepare to bring investigational LUXTURNA to patients in the U.S. and Europe," said John Furey, chief operating officer of Spark Therapeutics. "For the first time, these individuals, who eventually will progress to complete blindness, have hope for a potential treatment option that may restore their vision. We remain steadfast in our commitment to bring this important investigational therapy to patients in the EU with vision loss due to Leber congenital amaurosis or retinitis pigmentosa caused by confirmed biallelic RPE65 mutations."
Clinical Trial Overview of
The safety and efficacy of LUXTURNA were assessed in two open-label Phase 1 trials, which continue to follow participants who received LUXTURNA between 2007 and 2012, and one open-label, randomized, controlled Phase 3 trial. Following the one-year control period of the Phase 3 study, all control participants elected to cross over and received LUXTURNA; long-term safety and efficacy continue to be assessed in the Phase 3 participants who received LUXTURNA between 2013 and 2015. The clinical trial program included 41 participants with vision loss aged four to 44 at the time of first administration. Confirmed biallelic RPE65 mutations and the presence of sufficient viable retinal cells were established in all participants.
LUXTURNA Phase 3 clinical trial data, including data from the intent-to-treat population of all randomized participants through the one-year time point, were published in The Lancet. Results reported in The Lancet showed a statistically significant and clinically meaningful difference between intervention (n=21) and control participants (n=10) at one year, per the clinical trial's primary endpoint, mean bilateral multi-luminance mobility testing (MLMT) change score (difference of 1.6; 95% CI, 0.72, 2.41; p =0.0013). In addition, participants who received LUXTURNA showed a marked difference compared to control participants across the first two secondary endpoints: full-field light sensitivity threshold (FST) testing averaged over both eyes ( p =0.0004) and the mobility test change score for the first injected eye ( p =0.0005). A third secondary endpoint, the change in visual acuity (VA) averaged over both eyes, was not statistically significant between intervention and control participants ( p =0.17).
On average, participants in the original Phase 3 intervention group maintained functional gains observed by the day-30 visit through at least two years, as measured by MLMT and FST. The more than 100-fold (or greater than two log units) average improvement in FST testing observed in the original intervention group at one year, similarly, was maintained through at least two years.
In continuation of the trial to include crossover of the control group to receive LUXTURNA, 93 percent (27 of 29) of all Phase 3 trial participants injected with LUXTURNA saw a gain of functional vision as assessed by bilateral MLMT over the follow-up period of at least one year from administration of LUXTURNA to each eye. Additionally, 72 percent (21 of 29) of all Phase 3 trial participants receiving LUXTURNA successfully completed MLMT at the lowest light level evaluated (1 lux) at one year.
Data from a cohort of the Phase 1 clinical trial, in which investigational LUXTURNA was administered to the contralateral, or second previously uninjected eye, showed mean improvements in functional vision and visual function. These improvements were maintained through at least three years, as measured by both MLMT and FST testing. This cohort of participants (n=8) received the same dose of LUXTURNA that was administered in the Phase 3 trial and would have met the Phase 3 eligibility criteria.
No serious adverse events (SAEs) associated with LUXTURNA or deleterious immune responses have been observed. Two ocular SAEs were reported in the clinical program. There was one SAE related to the surgical procedure in one eye of a Phase 3 participant, in which there was foveal thinning and a sustained reduction in VA. One additional ocular SAE was reported in one eye of a Phase 1 participant in which the treatment for bacterial endophthalmitis led to elevated intraocular pressure and subsequent optic atrophy. There were three non-serious AEs of retinal deposits (subretinal precipitate) in three participants (three eyes) that were considered to be related to LUXTURNA. All three of these events were mild in intensity, transient in nature and resolved without consequences. The most common adverse reactions related to LUXTURNA reported in 10 percent or greater of the combined Phase 1 and Phase 3 trial participants included conjunctival hyperemia, cataract, intraocular pressure increased, and retinal tear.
-mediated Inherited Retinal Disease (IRD)
Inherited retinal diseases (also known as inherited retinal dystrophies) are a group of rare blinding conditions caused by one of more than 220 different genes. People living with IRD due to biallelic RPE65 gene mutations often experience night blindness (nyctalopia) due to decreased light sensitivity in childhood or early adulthood and involuntary back-and-forth eye movements (nystagmus). As the disease progresses, individuals may experience loss in their peripheral vision, developing tunnel vision, and eventually, they may lose their central vision as well, resulting in total blindness. Independent navigation becomes severely limited, and vision-dependent activities of daily living are impaired. There are currently no approved pharmacologic treatment options for IRD due to biallelic RPE65 gene mutations.
About Gene Therapy
Gene therapy is an investigational approach to treat or prevent genetic disease by seeking to augment, replace or suppress one or more mutated genes with functional copies. It addresses the root cause of an inherited disease by enabling the body to produce a protein or proteins necessary to restore health or to stop making a harmful protein or proteins, with the potential of bringing back function in the diseased cells and slowing disease progression. To deliver the functional gene into the cell, a vector is used to transport the desired gene and is delivered either intravenously (IV) or injected into specific tissue. The goal is to enable, through the one-time administration of gene therapy, a lasting therapeutic effect.
About Spark Therapeutics
Spark Therapeutics, a fully integrated company, strives to challenge the inevitability of genetic disease by discovering, developing, and delivering gene therapies that address inherited retinal diseases (IRDs), neurodegenerative diseases, as well as diseases that can be addressed by targeting the liver. Our validated platform has successfully delivered proof-of-concept data with investigational gene therapies in the retina and liver. Our most advanced investigational candidate, with proposed trade name LUXTURNA(TM) (voretigene neparvovec), is currently under Priority Review with FDA for the treatment of biallelic RPE65 -mediated IRD and has been designated as a drug for a rare pediatric disease. It previously received breakthrough therapy and orphan product designations from FDA and orphan product designations from the European Medicines Agency (EMA). The pipeline also includes SPK-7001 in a Phase 1/2 trial for choroideremia, and two hemophilia development programs: SPK-9001 (which also has received both breakthrough therapy and orphan product designations by FDA, and access to the PRIority MEdicines (PRIME) Program by the EMA) in a Phase 1/2 trial for hemophilia B being developed in collaboration with Pfizer; and SPK-8011 , in a Phase 1/2 trial for hemophilia A to which Spark Therapeutics retains global commercialization rights. For more information, visit www.sparktx.com.
Cautionary note on forward-looking statements
This release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995, including statements regarding the company's product candidate LUXTURNA(TM) (voretigene neparvovec). Any forward-looking statements are based on management's current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in, or implied by, such forward-looking statements. These risks and uncertainties include, but are not limited to, the risk that: (i) our BLA submitted for LUXTURNA to the FDA may not be approved; (ii) our MAA submitted for LUXTURNA to the EMA may not be validated or approved; (iii) the data from our Phase 3 clinical trial of LUXTURNA may not support US labeling for all biallelic RPE65 mutations other than Leber congenital amaurosis (LCA) or retinitis pigmentosa (RP); and (iv) the improvements in functional vision demonstrated by LUXTURNA in our clinical trials may not be sustained over extended periods of time. For a discussion of other risks and uncertainties, and other important factors, any of which could cause our actual results to differ from those contained in the forward-looking statements, see the "Risk Factors" section, as well as discussions of potential risks, uncertainties and other important factors, in our Annual Report on Form 10-K, our Quarterly Reports on Form 10-Q and other filings we make with the Securities and Exchange Commission. All information in this press release is as of the date of the release, and Spark undertakes no duty to update this information unless required by law.
Investor Relations Contact:
Monique da Silva
The issuer of this announcement warrants that they are solely responsible for the content, accuracy and originality of the information contained therein.
Source: Spark Therapeutics, Inc. via Globenewswire
One Liberty Plaza - 165 Broadway
NY 10006 New York
GlobeNewswire is one of the world's largest newswire distribution networks, specializing in the delivery of corporate press releases financial disclosures and multimedia content to the media, investment community, individual investors and the general public.
Følg saker fra GlobeNewswire
Registrer deg med din epostadresse under for å få de nyeste sakene fra GlobeNewswire på epost fortløpende. Du kan melde deg av når som helst.
Siste saker fra GlobeNewswire
BrandSafway™ Announces Acquisition of Century Elevators22.2.2019 20:00:00 | Pressemelding
Enhances motorized access capabilities in industrial and commercial construction markets throughout the Gulf Coast and beyond Kennesaw, Georgia, USA, Feb. 22, 2019 (GLOBE NEWSWIRE) -- KENNESAW, Georgia; February 22, 2019 – In a move to expand its motorized capabilities and offer a full suite of access technologies throughout the Gulf Coast, Brand Safway is pleased to announce the acquisition of Century Elevators, effective February 22, 2019. The leading rack and pinion elevator specialist in North America, Century Elevators provides construction and industrial elevators, material hoists, and transport platforms in the Gulf Coast region and beyond. Century Elevators is also the exclusive distributor of PEGA Hoist Ltd. products throughout North America and Böcker Maschinenwerke GmbH (Boecker) equipment in the United States. “Century Elevators is a very welcome addition to BrandSafway,” said Dave Witsken, president of Energy and Industrial at BrandSafway. “With the outstanding experience
Open Compute Project Announces Updated Market Forecast22.2.2019 16:39:00 | Pressemelding
Open Source Foundation Exceeds 2018 Forecast for Non-Board Member Adoption, Tops $2.56 billion and expected to surpass $10 billion by 2022. Austin, Texas, Feb. 22, 2019 (GLOBE NEWSWIRE) -- The Open Compute Project Foundation (OCP) announces today the high level results of a follow up assessment of the market impact of the Open Compute Project worldwide. For a second year, OCP has engaged IHS Markit, a world leader in critical information, analytics and solutions, to determine the adoption and impact of OCP gear in the technology industry. Since its inception, OCP has worked to drive innovation in and around the data center industry, bringing together thousands of engineers from nearly two hundred member organizations. The demands on the modern datacenter continue to expand with the growth of IOT, security and edge computing, as well as increasing energy consumption requirements. IHS Markit interviewed OCP members, suppliers and service providers, as well as incorporated their own in-de
Magna Announces Fourth Quarter and 2018 Results and Raises Quarterly Cash Dividend by 11%22.2.2019 11:00:00 | Pressemelding
Fourth Quarter 2018 Highlights Record fourth quarter sales of $10.1 billion up 5% from the fourth quarter of 2017 Cash from operations of $1.6 billion Returned $585 million to shareholders through share repurchases and dividends Raised quarterly cash dividend by 11% to $0.365 per share Full Year 2018 Highlights Record sales of $40.8 billion, up 12% from 2017 Record diluted earnings per share of $6.61, an increase of 13% Record cash from operations of $3.7 billion Returned approximately $2.3 billion to shareholders through share repurchases and dividends AURORA, Ontario, Feb. 22, 2019 (GLOBE NEWSWIRE) -- Magna International Inc. (TSX: MG; NYSE: MGA) today reported financial results for the fourth quarter and year ended December 31, 2018. THREE MONTHS ENDED DECEMBER 31, YEAR ENDED DECEMBER 31, 2018 2017 (2) 2018 2017 (2) Reported Sales $ 10,137 $ 9,684 $ 40,827 $ 36,588 Income from operations before income taxes $ 607 $ 765 $ 2,951 $ 2,985 Net income attributable to Magna International I
IMImobile announces integration of WhatsApp Business solution into its enterprise cloud communications platform IMIconnect22.2.2019 10:37:00 | Pressemelding
The WhatsApp Business solution will be made available across its enterprise cloud communications platform IMIconnect allowing businesses to launch business-to-consumer communications on WhatsApp LONDON, Feb. 22, 2019 (GLOBE NEWSWIRE) -- Global cloud communications software and solutions provider IMImobile PLC, today announced the integration of the WhatsApp Business solution into its enterprise cloud communications platform IMIconnect. The WhatsApp Business solution enables businesses to connect with over 1.5 billion users in a simple, reliable, and private way across 180 countries worldwide. As a WhatsApp Business solution provider, the IMIconnect platform will enable enterprises to seamlessly integrate the WhatsApp Business solution into their customer communications strategies, and drive engagement through intelligent and context-aware messaging. “We are excited to announce the integration of the WhatsApp Business solution today in our IMIconnect platform. We understand that today’s
Nordic Innovators Cloudstreet and Domos Partner to Deliver an End-to-End, Application-Aware 5G Experience to the Home22.2.2019 09:00:00 | Pressemelding
Cloudstreet’s carrier-grade API and Network Slicing platform with Domos’ Machine Learning Solution for home networks delivers intelligent connectivity out of the box Barcelona, Spain., Feb. 22, 2019 (GLOBE NEWSWIRE) -- Finland’s Cloudstreet, the US-patented Network Slicing Company, and Norway’s Domos, a leader in Machine Learning technologies for the smart home, are pleased to announce that they have teamed up to create the industry’s first end-to-end, intelligent application and context-aware network slicing solution for home networks. The solution will be on display February 25-28 at the Mobile World Congress in Barcelona in Hall 5, stand 5C41. A perfect example of MWC’s aspirational theme, “Intelligent Connectivity”, the solution closes the loop on delivering a 5G experience to fixed wireless home networks. The combined technologies solve two key, last-mile problems that have plagued mobile-enabled home networks: 1) How to build application-awareness into the network without comprom
General Electric Company: Doc re. GE Files Form 8-K22.2.2019 08:00:00 | Pressemelding
FAIRFIELD, Conn., Feb. 22, 2019 (GLOBE NEWSWIRE) -- Company General Electric Company ISIN US3696041033 Symbol London: GEC | Paris: GNE Headline Doc re: GE files Form 8-K February 21, 2019 On February 21, 2019, General Electric Company (the "Company") filed a Form 8-K with the U.S. Securities and Exchange Commission ("SEC"), which has been submitted to RNS. It is also available on the SEC's website at http://www.sec.gov and on the Company's website at https://www.ge.com/investor-relations/events-reports. http://www.rns-pdf.londonstockexchange.com/rns/8048Q_1-2019-2-21.pdf CONTACT: GE Jennifer Erickson +001 646 682 5620 email@example.com This information is provided by RNS, the news service of the London Stock Exchange. RNS is approved by the Financial Conduct Authority to act as a Primary Information Provider in the United Kingdom. Terms and conditions relating to the use and distribution of this information may apply. For further information, please contact firstname.lastname@example.org or visit