Latest news with #Qalsody
Medscape
30-07-2025
- Health
- Medscape
MHRA Approves First Targeted Drug for SOD1-ALS
The Medicines and Healthcare products Regulatory Agency (MHRA) has approved tofersen (Qalsody, Biogen) for treating amyotrophic lateral sclerosis (ALS) in adults with mutations in the superoxide dismutase 1 (SOD1) gene, also known as SOD1-ALS. The approval makes tofersen the first targeted therapy in the UK against this rare form of motor neurone disease (MND) with a genetic basis. A Rare but Aggressive Condition SOD1-ALS causes the body to produce a misfolded, toxic version of the SOD1 protein. This triggers motor neuron degeneration in the brain and spinal cord. Patients experience progressive muscle weakness, atrophy, respiratory decline, and eventual paralysis and death. SOD1 mutations are responsible for approximately 2% of all ALS cases globally. Over 200 distinct mutations in SOD1 have been identified, each contributing to a wide range of disease progression rates. This variability highlights the importance of developing targeted therapies for SOD1-ALS. How Tofersen Works Tofersen is an antisense oligonucleotide that binds to SOD1 mRNA, promoting its degradation through an RNase H-mediated mechanism. This reduces the production of the toxic SOD1 protein. The drug is administered via intrathecal injection through a lumbar puncture at scheduled intervals, under the supervision of trained healthcare professionals. Success in Clinical Trials The approval was supported by data from the phase 3 VALOR study and its open-label extension (OLE). In the VALOR trial, 108 adults with SOD1-ALS were treated with either intrathecal tofersen (n=72) or placebo (n=36) for 28 weeks. The primary endpoint, the ALSFRS-R score — which assesses bulbar, motor, and respiratory functions — showed a positive trend with tofersen, although the difference was not significant. However, tofersen significantly reduced biomarkers of neurodegeneration, including cerebrospinal fluid (CSF) SOD1 protein levels and plasma neurofilament light chain (NfL) concentrations. By week 28, plasma NfL levels decreased by 55% in the tofersen group, compared to a 12% increase with placebo. Clinical trends suggested improvements in respiratory function, muscle strength, and quality of life. These effects were more pronounced with earlier treatment and during the OLE phase. Safety and Tolerability The most common side effects in tofersen-treated patients included: Back, muscle, and joint pain Fatigue Fever Raised CSF protein levels or white blood cell counts These reactions were generally mild to moderate in severity. Further information, including the Patient Information Leaflet and Summary of Product Characteristics, will be available on the MHRA website within 7 days of approval.
Yahoo
23-06-2025
- Health
- Yahoo
Amyotrophic Lateral Sclerosis Treatment Market To Observe Growth at a CAGR of 6.1% by 2033
The global amyotrophic lateral sclerosis treatment market is undergoing gradual yet impactful transformation, driven by advancements in neurodegenerative disease research, gene-targeted therapies, and biomarkers innovation. Emerging therapeutic approaches, including antisense oligonucleotides and monoclonal antibodies, are enhancing precision treatment for genetically defined amyotrophic lateral sclerosis (ALS) subtypes, such as SOD1-linked ALS. Although ALS remains a rare condition, with an incidence of about 2 per 100,000 annually, it is receiving increased attention due to heightened awareness, improved diagnostic capabilities, and robust investment in R&D. The evolving treatment landscape is also benefiting from a growing emphasis on personalized medicine, which is reshaping disease management strategies for this devastating condition. Chicago, June 23, 2025 (GLOBE NEWSWIRE) -- The global amyotrophic lateral sclerosis treatment market is projected to grow from USD 724.5 million in 2024 to USD 1,234.5 million by 2033, reflecting a CAGR of 6.1% over the forecast period. This moderate yet steady expansion is supported by a rising aging population, advancements in disease-modifying therapies, and increasing prevalence rates. Although curative therapies remain elusive, the pipeline is increasingly populated with targeted investigational drugs, including small molecules, peptides, and biologics. Institutions and regulatory agencies are placing a growing focus on biomarker validation to accelerate clinical development and improve treatment outcomes. The recognition of biomarkers such as neurofilament light chain (NfL) as indicators of therapeutic response marks a pivotal step forward in streamlining ALS drug development. Download Free Sample Pages: The rising incidence of amyotrophic lateral sclerosis, particularly among adults aged 60 to 79, underscores the growing healthcare burden. In the U.S., the National ALS Registry estimated over 32,800 cases in 2022, with projections nearing 36,300 by 2030. The progressive nature and high mortality rate of ALS necessitate rapid intervention and proactive management. The predominance of sporadic ALS, comprising 90% of all cases, presents challenges in early detection and treatment targeting. Conversely, familial ALS, which accounts for roughly 10%, presents distinct genetic markers such as C9ORF72 and SOD1 mutations, offering a clearer path for precision therapies like Qalsody (Tofersen), which received FDA approval in 2023. Innovative drug approvals and clinical research are driving optimism. Treatments such as Riluzole, Edaravone, and more recently, gene-targeted agents like Tofersen and AMX0035, have brought incremental benefits. However, setbacks remains, for example; AMX0035 (Relyvrio) was withdrawn in 2024 following unsatisfactory phase III results. Nonetheless, therapeutic progress continues, with a growing number of trials exploring novel approaches, including stem cell-based interventions and gene editing technologies. The National Institutes of Health (NIH) has committed substantial funding, including a $25 million investment over five years, to accelerate ALS research and drug discovery efforts. Despite these advances, the amyotrophic lateral sclerosis treatment market continues to face notable challenges. The high cost of care, particularly in late-stage disease, imposes a significant financial burden. Treatment expenses in the U.S. can exceed $120,000 annually as the disease progresses, compounded by delayed diagnoses and complex care requirements. Furthermore, innovative gene therapies while promising carry a high price tags, as seen with Qalsody, which costs over $16,000 per 15 mL dose. Nevertheless, the amyotrophic lateral sclerosis treatment market holds promising potential, bolstered by biomarker innovation, gene-specific therapies, and a commitment to earlier diagnosis and intervention. With increasing efforts from academic research centers, pharmaceutical companies, and patient advocacy groups, the field is gradually shifting from symptomatic care toward disease-modifying approaches. As regulatory pathways evolve and precision medicine takes hold, the market is expected to offer more effective and personalized treatments, transforming the outlook for patients living with amyotrophic lateral sclerosis (ALS).Market Forecast (2033) USD 1,234.5 million CAGR 6.1% Top Driver Increasing Incidence of Amyotrophic Lateral Sclerosis (ALS) Among the Aging Population Top Trend Emerging Biomarkers in Amyotrophic Lateral Sclerosis Treatment Top Challenge Elevated Cost of Treating Amyotrophic Lateral Sclerosis (ALS) Biomarker Innovation: Advancing Diagnostic Precision and Accelerating ALS Therapeutics Rapid strides in biomarker research are transforming the treatment paradigm for amyotrophic lateral sclerosis (ALS), ushering in a new era of precision diagnostics and targeted therapeutic strategies. Historically constrained by delayed diagnoses and limited tools for tracking disease progression, the ALS field is now benefitting from biomarker technologies that enhance early detection, improve patient monitoring, and streamline the development of novel therapies. A pivotal breakthrough came in 2023 when the U.S. Food and Drug Administration (FDA) granted accelerated approval to Tofersen (Qalsody), establishing neurofilament light chain (NfL) as a qualified response biomarker. Clinical data revealed that reductions in NfL were indicative of slower neurodegeneration, validating its role in assessing treatment effectiveness. Building on this momentum, researchers are expanding their focus to identify other biomarkers, including abnormal forms of the TDP-43 protein, strongly associated with ALS pathology. Early detection of these proteins in cerebrospinal fluid or blood may enable real-time disease monitoring and inform more individualized treatment plans. Public-sector institutions are playing a key role in driving this progress. The National Institute of Neurological Disorders and Stroke (NINDS) is actively funding initiatives aimed at validating emerging biomarkers, particularly to shorten diagnostic timelines for patients with atypical or early-stage ALS. These efforts are critical to improving prognostic accuracy and tailoring therapeutic approaches to each patient's disease trajectory. As biomarker science continues to evolve, its integration into amyotrophic lateral sclerosis treatment and research is poised to deliver meaningful clinical and commercial impact. From optimizing trial design to facilitating faster regulatory approvals, biomarkers are redefining how amyotrophic lateral sclerosis (ALS) is diagnosed and managed moving the field closer to a future of earlier intervention, personalized care, and more effective therapies Global Amyotrophic Lateral Sclerosis Treatment Market: Regional Drivers Shaping the Future of Neuromuscular Care The global amyotrophic lateral sclerosis treatment market is segmented into North America, South America, Europe, Asia-Pacific, and the Middle East & Africa, with North America maintaining a dominant position. In the United States alone, an estimated 30,000 individuals are currently living with ALS, underscoring the urgent need for effective therapeutic options. The region leads in research and development, with most ALS medications receiving initial regulatory approval there. Key organizations like the ALS Association play a pivotal role in advancing treatment efforts, supporting a wide range of initiatives including grant-funded stem cell therapy programs. Enhanced insurance coverage further facilitates access to care, mitigating the financial burden of high treatment costs. Additionally, comprehensive support systems such as the ALS Network offer a wide array of services, from equipment loans and clinic access to benefit assistance, advocacy, and peer support groups, ensuring holistic care for patients and caregivers. Canada also plays an important role, with the Canadian Neuromuscular Disease Registry (CNDR) collecting nationwide clinical data to improve care standards and treatment accessibility. Building on the foundations established in North America, Europe has emerged as a strong contributor to ALS research and patient care. Collaborative efforts such as the ENCALS and TRICALS work with more than 60 centers across the continent to expand clinical trial availability, though currently, less than 5% of amyotrophic lateral sclerosis (ALS) patients in Europe participate in such studies. These efforts reflect a regional commitment to improving early access to experimental therapies and fostering cross-border collaboration in amyotrophic lateral sclerosis (ALS) care. The momentum continues to build in the Asia-Pacific region, where countries are increasingly investing in infrastructure and professional training. Japan is establishing dedicated multidisciplinary ALS centers, while emerging economies like India are making notable progress. In 2023/2024, over 550 Indian physicians received certification through Advanced Life Support (ALS) programs delivered in partnership with the European Resuscitation Council (ERC). These internationally accredited training initiatives are elevating standards of care and expanding clinical capacity across the region. While advancements are more gradual in the Middle East and Africa, the region is showing signs of long-term potential. With healthcare infrastructure and resources still in development, increasing attention to awareness-building, professional training, and international cooperation indicates a growing foundation for future market expansion. As these efforts mature, the region may play a more prominent role in the global amyotrophic lateral sclerosis treatment landscape. Modify Report as Per Requirements: Recent Advancements and Key Players in the Amyotrophic Lateral Sclerosis Treatment Market Several prominent players, including MediciNova, Mitsubishi Tanabe Pharma, Ionis Pharmaceuticals, Alector, Sanofi, ITF Pharma, Aquisitive Therapeutics, GSK, Denali Therapeutics, and BrainStorm Cell Therapeutics, are actively shaping the future of amyotrophic lateral sclerosis treatment through advancements in neurodegenerative research and synaptic regeneration. These companies are driving forward clinical development, regulatory progress, and therapeutic innovation in the amyotrophic lateral sclerosis treatment landscape. In a significant regulatory milestone, on June 3rd, 2025, Spinogenix announced that the European Medicines Agency (EMA) granted Orphan Drug Designation (ODD) to its lead candidate, SPG302, for the treatment of amyotrophic lateral sclerosis (ALS). This followed the recent completion of a Phase 2 clinical trial in Australia (NCT06903286), highlighting SPG302's potential as the first synaptic regenerative therapy for ALS. Further advancing ALS research, Transposon Therapeutics announced on May 28th, 2025, positive Phase 2 results for its investigational therapy TPN-101 in C9orf72-related ALS, showing reductions in neurofilament light chain (NfL) and interleukin-6 (IL-6) levels. TPN-101 is slated to enter the HEALEY ALS Platform Trial in Q4 2025, signaling continued momentum in adaptive trial designs. Similarly, on April 7th, 2025, Immunity Pharma reported positive Phase 2a clinical trial results for its investigational amyotrophic lateral sclerosis treatment, IPL344. The data showed that IPL344 significantly slowed the progression of ALS, as measured by the ALSFRS-R, by 58–64% compared to a matched control group from the PRO-ACT database. Additionally, treatment with IPL344 led to a 27% reduction in neurofilament light chain (NfL) levels after just two months, suggesting a potential disease-modifying effect. In a key regional development, the Japanese Ministry of Health, Labor and Welfare approved QALSODY in December 2024, expanding global access to this innovative therapy and underscoring Japan's regulatory support for amyotrophic lateral sclerosis treatments. These advancements underscore the rapid progress in amyotrophic lateral sclerosis drug development, driven by novel mechanisms of action and supportive global regulatory pathways. As leading companies continue to invest in clinical research and therapeutic innovation, the ALS treatment landscape is poised for transformation, offering renewed hope to patients worldwide. Future Outlook: Evolving Amyotrophic Lateral Sclerosis Treatments and the Rise of Regenerative Approaches The future of amyotrophic lateral sclerosis treatment is poised for significant change, with ongoing advancements in genetic therapies and an early promise in stem cell research. While current market leaders like Riluzole, Edaravone, and Qalsody continue to support disease management; high treatment costs and limited efficacy underscore the urgent need for more effective, accessible options. Stem cell therapies, particularly those involving mesenchymal stem cells (MSCs), remain in the research phase but are showing encouraging results. In 2025, DVCStem reported that MSCs may help delay onset, slow progression, and extend lifespan in ALS patients. Similarly, Mitsubishi Chemical's CL2020 completed Phase 2 trials with strong safety outcomes, reinforcing confidence in cell-based interventions. Looking ahead, the amyotrophic lateral sclerosis treatment landscape is expected to shift toward more personalized, regenerative, and combination-based approaches. As clinical trials advance and GMP-certified manufacturing expands, stem cell therapies may become a viable addition to standard care. By 2030, greater integration of genetic targeting, cellular therapies, and streamlined delivery models could redefine ALS management and offer new hope to patients worldwide. Key Competitors Medicinova Mitsubishi Tanabe Pharma Ionis Alector Sanofi ITF Pharma Aquisitive Therapuetice GSK Denali Therapuetics BrainStrom Segmentation of Amyotrophic Lateral Sclerosis Treatment Market By Treatment Type Medication Therapy Riluzole Radicava Qalsody Stem Cell Therapy By Distribution Channel Hospital & Clinics Pharmacies By Region North America South America Europe Asia-Pacific Middle East and Africa Have Questions? Reach Out Before Buying: About Astute Analytica Astute Analytica is a global market research and advisory firm providing data-driven insights across industries such as technology, healthcare, chemicals, semiconductors, FMCG, and more. We publish multiple reports daily, equipping businesses with the intelligence they need to navigate market trends, emerging opportunities, competitive landscapes, and technological advancements. With a team of experienced business analysts, economists, and industry experts, we deliver accurate, in-depth, and actionable research tailored to meet the strategic needs of our clients. At Astute Analytica, our clients come first, and we are committed to delivering cost-effective, high-value research solutions that drive success in an evolving marketplace. 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CBC
11-03-2025
- Health
- CBC
Regina woman is living proof of 'breakthrough' ALS drug now approved in Canada
Social Sharing A Regina mother who was one of the first ALS patients to test a new treatment is now celebrating its accelerated approval in Canada for adults with an ultra-rare form of the disease. Paula Trefiak's remarkable experience on tofersen, a medication sold under the brand name Qalsody, is reflective of why researchers are calling the drug a breakthrough. "My life has completely changed. I am actually looking forward to retirement now. I never thought I would ever get to retirement," Trefiak, 43, told CBC News. People diagnosed with amyotrophic lateral sclerosis (ALS), a fatal neurodegenerative disorder also known as Lou Gehrig's disease, have a life expectancy of two to five years. They become trapped in their body, losing the ability to move, speak, chew and eventually breathe. Health Canada confirms it has granted conditional approval to Qalsody to treat adults with ALS in cases linked to a mutation in the superoxide dismutase 1 (SOD1) gene. Ottawa expedited access to the drug under a program that recognizes it would be unethical to withhold it from patients, but pharmaceutical company Biogen must still complete additional clinical trials. The drug is not yet covered under public or private drug programs. Family history Trefiak, 43, realized she had a pretty good chance of developing ALS when her father was diagnosed with the disease in 2001. She can count 26 people in her family who have died from the disease in her lifetime. Most ALS cases are sporadic, but a small fraction are genetic. About three per cent of all ALS cases are linked to a mutation in the SOD1 gene. Despite her family history, Trefiak said doctors were reluctant to send her for genetic testing when she was in her early 20s. She recounts one doctor telling her, "You're too young to have it. And there's no cure. There's no treatment. So it's best not to find out." By 2010, her muscles began to cramp for hours and Trefiak felt certain she had inherited the gene variant. She was diagnosed with ALS in February 2016, at the age of 34. Her prognosis was dire. There were no treatments available. "It was absolutely devastating. I had three young children, and to know that I was going to lose my ability to hug them, to kiss them, and that they were going to lose their childhoods becoming my caregivers," she said. Nine months later, she got a phone call that gave her some hope. She was invited to the Neuro Hospital in Montreal to take part in a clinical trial designed for people with ALS caused by a mutation in the SOD1 gene. She believes she received the placebo in that clinical trial, but began the full dosage of 100 mg in early 2018, during the open label portion of the study. By then, involuntary muscle twitches in her face and lip were causing drooling and stuttering. She was too weak to continue her jobs as a fitness instructor and emergency medical responder, and had to give up on her schooling to become a dental hygienist because she couldn't hold the instruments any longer. Within nine months of taking the drug, many of the symptoms reversed. Clinical trials Dr. Angela Genge is a neurologist, director of the ALS Centre of Excellent at The Neuro in Montreal and lead investigator in the clinical trials. She said the medication actually modifies the disease itself and slows — even stops — the progression. "It is able to stop the disease in its tracks on a significant number of patients or people living with ALS," Genge said. "[Qalsody] is actually a huge breakthrough." Genge points to the fact that Paula Trefiak is still alive. "People are not dying who are successfully on the treatment," the neurologist said, emphasizing the drug is not a cure and that benefits only continue as long as the drug is taken. Today, Trefiak runs and dances. "I'm a ballet dancer. I couldn't even get up on my toes anymore and now I can hold myself up on with full body weight on one foot and I'm back wearing my three-inch heels again," she said. WATCH | Paula Trefiak recorded involuntary muscle twitching in her face in 2017. See how she has improved: Paula Trefiak recorded involuntary muscle twitching in her face in 2017. See how she improved 18 hours ago Duration 0:34 Regina ALS patient Paula Trefiak took part in clinical trials for a drug that has slowed the progression of the disease and reversed most of her symptoms. Exciting possibilities Dr. Genge said this treatment is already inspiring other research on therapies for forms of ALS not caused by SOD1, as well as the drug's impact on people who have the gene mutation but have yet to show symptoms. "So that we catch people at the very, very, very beginning, so they never actually develop anything significant and we can treat them right away," Genge said, noting blood tests can reveal when the disease activates. That's also exciting for Trefiak. She has three children who may have inherited the gene mutation. This drug could stop them from developing ALS symptoms and help them live long lives.
