SPINRAZA® (nusinersen) Approved in the European Union
In conjunction with the SPINRAZA approval in the EU, Ionis earned a
"This is a landmark day for the European SMA community. The approval of SPINRAZA to treat a broad range of patients with SMA provides patients currently living with SMA hope for disease stabilization or improvement. Further, I am encouraged that the approval of SPINRAZA may demonstrate the potential of other antisense oligonucleotides to treat more neurodegenerative disorders,"
The approval of SPINRAZA was primarily based on results from two pivotal multicenter, controlled studies, including end of study data from ENDEAR (infantile-onset SMA) and an interim analysis of CHERISH (later-onset SMA), both of which demonstrated the clinically meaningful efficacy and favorable benefit-risk profile of SPINRAZA. The approval was also supported by results from open-label studies in pre-symptomatic and symptomatic individuals with, or most likely to develop, Types 1, 2 or 3 SMA.
"The approval of SPINRAZA by the
"We would like to convey our gratitude to the patients, families, physicians and their teams in the EU who participated in our clinical trials. Their support and dedication have been critical in achieving this important milestone," said
Biogen licensed the global rights to develop, manufacture and commercialize SPINRAZA from
ABOUT ENDEAR and CHERISH STUDIES
In the ENDEAR end of study analysis, a statistically significant greater percentage of patients achieved the definition of motor milestone responder in the SPINRAZA group (51%) compared to the sham-control group (0%) (p<0.0001). Some infants in the SPINRAZA group achieved motor milestones including full head control, ability to roll, sitting, and standing. Additionally, infants treated with SPINRAZA demonstrated a statistically significant reduction (47%) in the risk of death or permanent ventilation (p=0.0046).
In the CHERISH pre-specified interim analysis, there was a statistically significant and clinically meaningful improvement in motor function in children with later-onset SMA (most likely to develop Type 2 or Type 3) treated with SPINRAZA compared to untreated children. Improvements were measured by the Hammersmith Functional Motor Scale Expanded (HFMSE) and demonstrated a treatment difference of 5.9 points in the mean change from baseline to Month 15 in the HFMSE score (p=0.0000002). The HFMSE is a reliable and validated tool specifically designed to assess motor function in children with SMA. The Phase 3 end of study data were consistent with the interim analysis and presented at the
Spinal Muscular Atrophy (SMA) is characterized by loss of motor neurons in the spinal cord and lower brain stem, resulting in severe and progressive muscular atrophy and weakness. Ultimately, individuals with the most severe type of SMA can become paralyzed and have difficulty performing the basic functions of life, like breathing and swallowing.
Due to a loss of, or defect in the SMN1 gene, people with SMA do not produce enough survival motor neuron (SMN) protein, which is critical for the maintenance of motor neurons. The severity of SMA correlates with the amount of SMN protein. People with Type 1 SMA, the type that requires the most intensive and supportive care, produce very little SMN protein and do not achieve the ability to sit without support or live beyond two years without respiratory support. People with Type 2 and Type 3 produce greater amounts of SMN protein and have less severe, but still life-altering, forms of SMA.
ABOUT SPINRAZA (nusinersen)
SPINRAZA is being developed globally for the treatment of SMA.
SPINRAZA is an antisense oligonucleotide (ASO), using
SPINRAZA must be administered via intrathecal injection, which delivers therapies directly to the cerebrospinal fluid (CSF) around the spinal cord, where motor neurons degenerate in patients with SMA due to insufficient levels of SMN protein.
In 2016, in response to the urgent need for treatment for the most severely affected individuals living with SMA, Biogen sponsored one of the largest, pre-approval Expanded Access Programs (EAP) in rare disease free of charge. The EAP has successfully led to the initiation and ongoing treatment of more than 350 eligible individuals with infantile-onset SMA (most likely to develop Type 1) in 17 European countries.
SPINRAZA demonstrated a favorable benefit-risk profile. For SPINRAZA prescribing information in the EU, please visit http://www.ema.europa.eu/ema/.
Ionis is the leading company in RNA-targeted drug discovery and development focused on developing drugs for patients who have the highest unmet medical needs, such as those patients with severe and rare diseases. Using its proprietary antisense technology, Ionis has created a large pipeline of first-in-class or best-in-class drugs, with over three dozen drugs in development. SPINRAZA® (nusinersen) is a drug that has been approved in the U.S. for the treatment of spinal muscular atrophy (SMA) in pediatric and adult patients. Biogen is responsible for commercialization of SPINRAZA. Drugs that have successfully completed Phase 3 studies include volanesorsen, a drug Ionis is developing and plans to commercialize through its subsidiary,
IONIS' FORWARD-LOOKING STATEMENT
This press release includes forward-looking statements regarding Ionis' strategic relationship with Biogen and the development, activity, therapeutic potential, safety and commercialization of SPINRAZA. Any statement describing Ionis' goals, expectations, financial or other projections, intentions or beliefs is a forward-looking statement and should be considered an at-risk statement. Such statements are subject to certain risks and uncertainties, particularly those inherent in the process of discovering, developing and commercializing drugs that are safe and effective for use as human therapeutics, and in the endeavor of building a business around such drugs. Ionis' forward-looking statements also involve assumptions that, if they never materialize or prove correct, could cause its results to differ materially from those expressed or implied by such forward-looking statements. Although Ionis' forward-looking statements reflect the good faith judgment of its management, these statements are based only on facts and factors currently known by Ionis. As a result, you are cautioned not to rely on these forward-looking statements. These and other risks concerning Ionis' programs are described in additional detail in Ionis' annual report on Form 10-K for the year ended
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Ionis Pharmaceuticals Investor and Media Contact: D. Wade Walke, Ph.D., Vice President, Corporate Communications and Investor Relations, 760-603-2741