SMN2

Orphan designation: branaplam Treatment of spinal muscular atrophy, 16/04/2018 Withdrawn

Retrieved on: 
Thursday, April 18, 2024
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Orphan designation: branaplam Treatment of spinal muscular atrophy, 16/04/2018 Withdrawn

Key Points: 


Orphan designation: branaplam Treatment of spinal muscular atrophy, 16/04/2018 Withdrawn

New Biomarker Data Add Further Evidence Supporting the Potential Benefit of SPINRAZA® (nusinersen) in Infants and Toddlers with Unmet Clinical Needs after Gene Therapy

Retrieved on: 
Wednesday, March 6, 2024
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* The Phase 4 study evaluates clinical outcomes and safety following treatment with SPINRAZA over a 2-year period in infants and toddlers with spinal muscular atrophy (SMA) who have unmet clinical needs after treatment with Zolgensma® (onasemnogene abeparvovec).

Key Points: 
  • * The Phase 4 study evaluates clinical outcomes and safety following treatment with SPINRAZA over a 2-year period in infants and toddlers with spinal muscular atrophy (SMA) who have unmet clinical needs after treatment with Zolgensma® (onasemnogene abeparvovec).
  • The new data show that plasma neurofilament light chain (NfL) levels, an objective biomarker of axonal injury and neurodegeneration, were reduced in nearly all study participants treated with SPINRAZA.
  • These data will be presented at the 2024 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference (March 3-6, 2024).
  • “Our evolving understanding of gene therapy indicates there may be an opportunity for better outcomes,” said Crystal Proud, M.D., Pediatric Neurologist at Children’s Hospital of the King’s Daughters.

Human medicines European public assessment report (EPAR): Zolgensma, onasemnogene abeparvovec, Date of authorisation: 18/05/2020, Revision: 14, Status: Authorised

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Tuesday, January 2, 2024
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Human medicines European public assessment report (EPAR): Zolgensma, onasemnogene abeparvovec, Date of authorisation: 18/05/2020, Revision: 14, Status: Authorised

Key Points: 


Human medicines European public assessment report (EPAR): Zolgensma, onasemnogene abeparvovec, Date of authorisation: 18/05/2020, Revision: 14, Status: Authorised

ASURAGEN TO PRESENT NOVEL CLINICAL TESTING DATA AT AMP 2023 CONFERENCE

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Thursday, November 2, 2023
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MINNEAPOLIS , Nov. 2, 2023 /PRNewswire/ -- Bio-Techne Corporation (NASDAQ:TECH) today announced that Asuragen, a Bio-Techne brand, will present new data on clinical laboratory testing solutions related to cystic fibrosis variant detection, cancer monitoring, resolving conventionally inaccessible, disease-causing genes, and more at the upcoming meeting of the Association for Molecular Pathology (AMP), to be held November 14-18 in Salt Lake City, Utah. Attendees can learn about Asuragen's new products and innovations through a series of corporate workshops and poster presentations.

Key Points: 
  • Attendees can learn about Asuragen's new products and innovations through a series of corporate workshops and poster presentations.
  • "We look forward to sharing new data that reinforces our commitment to more equitable healthcare, comprehensive genetic coverage, and novel approaches to cancer monitoring and management."
  • In addition, Dr. Jordan Laser, Senior Director, Clinical and Medical Affairs for Bio-Techne's Diagnostics and Genomics Segment, will be recognized at the conference with the AMP Meritorious Service Award for his work to promote AMP as a member of the association.
  • AmplideX® to the Rescue: One easy-to-implement test workflow enabling repeat expansion resolution, copy number assessment, and highly multiplexed variant detection.

Royalty Pharma Announces Agreement to Purchase Up to $1.5 Billion of PTC Therapeutics’ Royalty on Evrysdi

Retrieved on: 
Thursday, October 19, 2023
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In 2022, Evrysdi generated sales of approximately CHF 1.1 billion ($1.2 billion), an increase of 87% at constant exchange rates versus the prior year.

Key Points: 
  • In 2022, Evrysdi generated sales of approximately CHF 1.1 billion ($1.2 billion), an increase of 87% at constant exchange rates versus the prior year.
  • Following the transaction announced today, in total, Royalty Pharma is increasing its ownership of the Evrysdi royalty from 43% to 81%, equating to a royalty of 6.5% to 13% (not including any exercise of future options by PTC or Royalty Pharma).
  • If PTC exercises fewer than three of these options, Royalty Pharma has the option to purchase 50% of the remaining PTC royalty for $250 million less royalties received until March 31, 2026.
  • Since 2020, Royalty Pharma has announced transactions of up to $12.8 billion, including up to $3.8 billion in 2023.

Majority of Newborn Babies With Spinal Muscular Atrophy (SMA) Treated With Genentech’s Evrysdi Able to Sit Independently After 1 Year of Treatment

Retrieved on: 
Wednesday, October 4, 2023
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“Evrysdi is the only non-invasive SMA treatment and can be used within hours of birth, potentially allowing these babies to sit, stand and walk, similar to healthy individuals,” said Levi Garraway, M.D., Ph.

Key Points: 
  • “Evrysdi is the only non-invasive SMA treatment and can be used within hours of birth, potentially allowing these babies to sit, stand and walk, similar to healthy individuals,” said Levi Garraway, M.D., Ph.
  • The primary efficacy population included babies with 2 SMN2 copies and a CMAP amplitude of ≥1.5 mV at baseline.
  • The majority of AEs were not considered treatment-related, and there were no deaths or AEs leading to withdrawal or treatment discontinuation.
  • The AEs observed in the RAINBOWFISH primary analysis are generally consistent with those AEs seen in other Evrysdi trials in SMA.

CHMP Recommends Evrysdi® for Babies Under Two Months Old with Spinal Muscular Atrophy

Retrieved on: 
Friday, July 21, 2023
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SOUTH PLAINFIELD, N.J., July 21, 2023 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced that the Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion for the extension of the Evrysdi® (risdiplam) marketing authorization to include infants under two months of age in the European Union (EU). A final decision regarding the approval is expected from the European Commission later this year.

Key Points: 
  • A final decision regarding the approval is expected from the European Commission later this year.
  • "The CHMP opinion represents another important milestone in the development of Evrysdi," said Matthew B. Klein, M.D., Chief Executive Officer, PTC Therapeutics.
  • "Presymptomatic babies are an important patient group that deserve access to oral, at-home administered treatments early in the disease."
  • Roche leads the clinical development of Evrysdi as part of a collaboration with the SMA Foundation and PTC Therapeutics.

New Data at Cure SMA Highlight Potential Benefit of SPINRAZA® (nusinersen) in Infants and Toddlers with Unmet Clinical Needs After Gene Therapy

Retrieved on: 
Friday, June 30, 2023
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(Nasdaq: BIIB) announced new SPINRAZA® (nusinersen) data aimed at answering critical questions for the spinal muscular atrophy (SMA) community.

Key Points: 
  • (Nasdaq: BIIB) announced new SPINRAZA® (nusinersen) data aimed at answering critical questions for the spinal muscular atrophy (SMA) community.
  • No new emerging safety concerns have been identified in enrolled participants who received SPINRAZA after Zolgensma.
  • “The RESPOND study has begun to characterize remaining unmet need in some SMA patients treated with Zolgensma whose outcomes have not met clinical expectations.
  • This summer, Alcyone plans to begin initial enrollment of the PIERRE study ( clinicaltrials.gov ), which will evaluate the safety and performance of ThecaFlex for the delivery of SPINRAZA in SMA patients.

Four-Year Follow-Up Data for Genentech’s Evrysdi Show Continued Increase in Number of Children With a Severe Form of Spinal ​Muscular ​Atrophy (SMA)​ Able to Sit, Stand and Walk

Retrieved on: 
Friday, June 30, 2023
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After four years of treatment with Evrysdi, many of the babies, now young children, continued to improve their ability to sit, stand and walk without support.

Key Points: 
  • After four years of treatment with Evrysdi, many of the babies, now young children, continued to improve their ability to sit, stand and walk without support.
  • All the Evrysdi-treated children who were alive at the time of the primary analysis were still alive at month 48.
  • Additionally, the majority of infants maintained their ability to feed by mouth and swallow up to month 48.
  • Genentech leads the clinical development of Evrysdi as part of a collaboration with the SMA Foundation and PTC Therapeutics.

Novartis shares Zolgensma long-term data demonstrating sustained durability up to 7.5 years post-dosing; 100% achievement of all assessed milestones in children treated prior to SMA symptom onset

Retrieved on: 
Sunday, March 19, 2023
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“These children now have an improved quality of life, vastly different from what would have been expected for them if they had not received treatment.

Key Points: 
  • “These children now have an improved quality of life, vastly different from what would have been expected for them if they had not received treatment.
  • Results from the IV cohort, which included 63 patients, demonstrated how a single administration of Zolgensma provided consistent, substantial and durable efficacy over time.
  • “Data from the LT-001 and LT-002 studies showed that, regardless of the patient’s symptomatic status at the time of treatment, Zolgensma IV is an effective and durable treatment option.
  • Nor can there be any guarantee that such products will be commercially successful in the future.