Orphan Drug Act of 1983

Global Rare Hemophilia Factors (I, II, VII) Market Size, Share & Trends Analysis Report 2023-2030 - ResearchAndMarkets.com

Retrieved on: 
Friday, October 27, 2023
Health, Genetics, FDA, Child, Plasma, Second Continental Congress, Orphan Drug Act of 1983, Health care, Haemophilia, ODA, Hand, Treatment, Congress, Prevalence, VII, Coagulation factor VII, Personal protective equipment, Coagulation, Infection, Quality of life, Growth, COVID-19, Factor VII deficiency, Patient, Diagnosis, Speciality chemicals, Pharmaceutical industry, Tisagenlecleucel, Safety, Cryoprecipitate

The "Rare Hemophilia Factors Market Size, Share & Trends Analysis Report By Type (Factor I, Factor II, Factor VII), By Treatment (Factor Concentrates, Fresh Frozen Plasma, Cryoprecipitate), By Region, And Segment Forecasts, 2023 - 2030" report has been added to ResearchAndMarkets.com's offering.

Key Points: 
  • The "Rare Hemophilia Factors Market Size, Share & Trends Analysis Report By Type (Factor I, Factor II, Factor VII), By Treatment (Factor Concentrates, Fresh Frozen Plasma, Cryoprecipitate), By Region, And Segment Forecasts, 2023 - 2030" report has been added to ResearchAndMarkets.com's offering.
  • The global rare hemophilia factors market is poised for substantial growth, targeting a USD 499.5 million market size by 2030, accompanied by a noteworthy compound annual growth rate (CAGR) of 6.9% from 2023 to 2030.
  • Prophylactic treatment is recommended for individuals with severe rare hemophilia factor disorders to prevent bleeding episodes and enhance their quality of life.
  • This intensified focus on patient safety measures is expected to have enduring benefits for rare hemophilia factor administration.

U.S. FDA AWARDS BOTH RARE PEDIATRIC DISEASE AND ORPHAN DRUG DESIGNATIONS TO HG204, A CRISPR RNA-EDITING THERAPY, FOR THE TREATMENT OF MECP2 DUPLICATION SYNDROME

Retrieved on: 
Tuesday, October 31, 2023
FDA, Ageing, Orphan Drug Act of 1983, Food, Rare, RNA, Sponsor, MDS, CRISPR, Greater China, Prevalence, MECP2, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Frasier, ODD, Speech, Neurodevelopmental disorder, Clinical trial, RNA editing, Messenger, MECP2 duplication syndrome, Therapy, Patient, GM, Pharmaceutical industry

"Although there is currently no clinical trial evaluating MDS, this is a sign of recognition for MDS and raises hopes for children and teens with this fatal, devastating disorder.

Key Points: 
  • "Although there is currently no clinical trial evaluating MDS, this is a sign of recognition for MDS and raises hopes for children and teens with this fatal, devastating disorder.
  • The FDA defines rare pediatric diseases as rare diseases (with fewer than 200,000 cases in the United States) that are serious or life threatening and primarily affect individuals aged under 18.
  • When the FDA awards rare pediatric disease designation (RPDD), the Sponsor of the trial firstly is granted a 6-months accelerated review of the drug candidate irrespective of indication, and which may be sold to another company.
  • "MDS is a rare and fatal childhood disease with the progressive of neurological regression such as loss of motor skills and speech.

U.S. FDA AWARDS BOTH RARE PEDIATRIC DISEASE AND ORPHAN DRUG DESIGNATIONS TO HG204, A CRISPR RNA-EDITING THERAPY, FOR THE TREATMENT OF MECP2 DUPLICATION SYNDROME

Retrieved on: 
Tuesday, October 31, 2023
FDA, Ageing, Orphan Drug Act of 1983, Food, Rare, RNA, Sponsor, MDS, CRISPR, Greater China, Prevalence, MECP2, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Frasier, ODD, Speech, Neurodevelopmental disorder, Clinical trial, RNA editing, Messenger, MECP2 duplication syndrome, Therapy, Patient, GM, Pharmaceutical industry

"Although there is currently no clinical trial evaluating MDS, this is a sign of recognition for MDS and raises hopes for children and teens with this fatal, devastating disorder.

Key Points: 
  • "Although there is currently no clinical trial evaluating MDS, this is a sign of recognition for MDS and raises hopes for children and teens with this fatal, devastating disorder.
  • The FDA defines rare pediatric diseases as rare diseases (with fewer than 200,000 cases in the United States) that are serious or life threatening and primarily affect individuals aged under 18.
  • When the FDA awards rare pediatric disease designation (RPDD), the Sponsor of the trial firstly is granted a 6-months accelerated review of the drug candidate irrespective of indication, and which may be sold to another company.
  • "MDS is a rare and fatal childhood disease with the progressive of neurological regression such as loss of motor skills and speech.

Sage Therapeutics Announces U.S. Food and Drug Administration Granted SAGE-718 Orphan Drug Designation for the Treatment of Huntington’s Disease

Retrieved on: 
Wednesday, October 18, 2023
Science, Neurology, Biotechnology, Research, Pharmaceutical, Health, FDA, Clinical Trials, AD, Orphan Drug Act of 1983, PD, Food, MCI, Rare, DSM-IV codes, HD, European Medicines Agency, Cognitive deficit, Deficit spending, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, ODD, Disease, Clinical trial, Marketing, Therapy, NMDA, Patient, Plasma protein binding, Pharmaceutical industry

Sage Therapeutics, Inc. (Nasdaq: SAGE), a biopharmaceutical company leading the way to create a world with better brain health, today announced the U.S. Food and Drug Administration (FDA) granted Orphan Drug Designation (ODD) to SAGE-718 for the treatment of Huntington’s disease (HD).

Key Points: 
  • Sage Therapeutics, Inc. (Nasdaq: SAGE), a biopharmaceutical company leading the way to create a world with better brain health, today announced the U.S. Food and Drug Administration (FDA) granted Orphan Drug Designation (ODD) to SAGE-718 for the treatment of Huntington’s disease (HD).
  • Cognitive impairment is one of the most underrecognized aspects of this disease,” said Laura Gault, M.D., Ph.D., Chief Medical Officer, Sage Therapeutics.
  • The approval of an orphan designation request does not alter the standard regulatory requirements and process for obtaining marketing approval.
  • SAGE-718 previously received Fast Track Designation from the FDA for HD, and orphan drug designation for HD by the European Medicines Agency.

Global Rare Hemophilia Factors Market Analysis Report 2023-2030 - Tailored Factor Concentrates Gain FDA Approval, Fueling Rare Hemophilia Factors Market Growth

Retrieved on: 
Tuesday, October 3, 2023
Orphan Drug Act of 1983, Fresh frozen plasma, Infection, Hand, Prevalence, Personal protective equipment, Health care, COVID-19, Quality of life, Haemophilia, Coagulation, Second Continental Congress, Congress, Plasma, ODA, VII, Patient, Child, Coagulation factor VII, Factor VII deficiency, Growth, Diagnosis, FDA, Treatment, Speciality chemicals, Pharmaceutical industry, Safety, Tisagenlecleucel

The global rare hemophilia factors market is poised for substantial growth, targeting a USD 499.5 million market size by 2030, accompanied by a noteworthy compound annual growth rate (CAGR) of 6.9% from 2023 to 2030.

Key Points: 
  • The global rare hemophilia factors market is poised for substantial growth, targeting a USD 499.5 million market size by 2030, accompanied by a noteworthy compound annual growth rate (CAGR) of 6.9% from 2023 to 2030.
  • The surge in the market is primarily attributed to the increased availability of tailored factor concentrates securing regulatory approvals, notably from the U.S. FDA.
  • Additionally, the rising adoption of prophylaxis treatment among patients is expected to be a significant growth driver in the coming years.
  • Prophylactic treatment is recommended for individuals with severe rare hemophilia factor disorders to prevent bleeding episodes and enhance their quality of life.

Dyne Therapeutics Receives FDA Orphan Drug Designation for DYNE-101 for the Treatment of Myotonic Dystrophy Type 1

Retrieved on: 
Wednesday, September 20, 2023
Orphan Drug Act of 1983, DYN, Therapy, DM1, Clinical trial, Plasma protein binding, Diagnosis, FDA, MPH, Second Half, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Dyne, Food, Safety, Pharmaceutical industry

WALTHAM, Mass., Sept. 20, 2023 (GLOBE NEWSWIRE) -- Dyne Therapeutics, Inc. (Nasdaq: DYN), a clinical-stage muscle disease company focused on advancing innovative life-transforming therapeutics for people living with genetically driven diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation for DYNE-101 for the treatment of myotonic dystrophy type 1 (DM1). DYNE-101 is being evaluated in the Phase 1/2 global ACHIEVE clinical trial with initial data on safety, tolerability and splicing from the multiple ascending dose, placebo-controlled portion of the trial anticipated in the second half of 2023.

Key Points: 
  • DYNE-101 is being evaluated in the Phase 1/2 global ACHIEVE clinical trial with initial data on safety, tolerability and splicing from the multiple ascending dose, placebo-controlled portion of the trial anticipated in the second half of 2023.
  • “Receiving FDA orphan drug designation for DYNE-101 underscores the importance of bringing new treatment options to people living with DM1, a rare, devastating disease with no approved therapies.
  • We believe DYNE-101 has the potential to be a transformative therapy which is why we designed the ACHIEVE trial to be registrational,” said Wildon Farwell, M.D., MPH, chief medical officer of Dyne.
  • Under the Orphan Drug Act, orphan drug designation qualifies a company for incentives, including tax credits, exemptions from certain FDA fees for clinical trials, and the potential for seven years of market exclusivity following drug approval.

From AI to Patient Advocacy: The Comprehensive Orphan Drugs and Rare Diseases Conference: London, United Kingdom - October 9-10, 2023

Retrieved on: 
Saturday, September 23, 2023
Orphan Drug Act of 1983, Infection, Regenerative medicine, ODA, AI, Rare, Drug development, Patient, Conference, Marketing, Pharmaceutical industry

DUBLIN, Sept. 22, 2023 /PRNewswire/ -- ResearchAndMarkets.com announces the 4th Annual Orphan Drugs and Rare Diseases Conference , scheduled for October 9-10 in London, UK.

Key Points: 
  • DUBLIN, Sept. 22, 2023 /PRNewswire/ -- ResearchAndMarkets.com announces the 4th Annual Orphan Drugs and Rare Diseases Conference , scheduled for October 9-10 in London, UK.
  • This prestigious event offers an in-depth exploration into the ever-evolving world of orphan drugs, post the landmark 1983 Orphan Drug Act (ODA).
  • The conference will spotlight novel therapies addressing the diverse and unique challenges of rare diseases, recognizing the vital importance of meeting the pressing needs of patients.
  • It also seeks to address the current challenges facing the field, including constrained resources, the severity of these diseases, restricted patient access, and the prevalent knowledge gap in the realm of rare diseases.

Pathways to Progress: How Orphan Drug Designation Sparks Biotech Transformation

Retrieved on: 
Wednesday, September 20, 2023
Atezolizumab, Diabetes, Neoplasm, Doctor of Philosophy, ODA, National library, Patient, GC, Disease, Prostate cancer, Carcinoma, CD47, Hope, Research, ACHL, Lung cancer, NLM, Orphan drug, Death, Drug development, Diagnosis, Growth, Rare, FDA, Legislation, Civil service commission, ODD, Roche, Orphan Drug Act of 1983, PharmaMar, Cancer, NSCLC, Treatment of lung cancer, ES, Therapy, Stomach, ORR, European Commission, File, United States National Library of Medicine, MedImmune, Inc. v. Genentech, Inc., Clinical trial, Woman, Lung, Fast track (FDA), SCLC, American Cancer Society, Achilles, Melanoma, OTCQX, Orphan, EC, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, FTD, RET, Food, Breast cancer, Pharmaceutical industry, Medical imaging, Genentech, Pembrolizumab

NEW YORK, Sept. 20, 2023 /PRNewswire/ -- More than 30 million people in the United States suffer with a rare disease; most of them are receiving no current treatment. Supporting the development and evaluation of new treatments, particularly for rare diseases, is a key priority for the U.S. Food & Drug Administration. As part of that process, the agency can grant Orphan Drug Designation (ODD) to a drug or biological product being developed to prevent, diagnose or treat a rare disease or condition. This designation is intended to spark innovation among biotech companies that are developing treatments for these patient populations, which by definition affect fewer than 200,000 people in the U.S., by providing incentives such as tax credits, user-fee exemptions and up to seven years of market exclusivity after FDA approval. Last month, Genprex Inc. (NASDAQ: GNPX) (Profile) was granted FDA orphan drug designation for its REQORSA(R) immunogene therapy in development for the treatment of small cell lung cancer (SCLC). The Phase 1/2 clinical trial, expected to dose the first patient in the fourth quarter of 2023, uses a combination of REQORSA and Genentech Inc.'s Tecentriq(R) as maintenance therapy in patients with extensive stage small cell lung cancer ("ES-SCLC") who did not develop tumor progression after receiving Tecentriq and chemotherapy as initial standard treatment. Genprex joins other companies — including Roche Holding AG ADR (OTCQX: RHHBY), Jazz Pharmaceuticals PLC (NASDAQ: JAZZ), ALX Oncology Holdings Inc. (NASDAQ: ALXO) and Achilles Therapeutics PLC (NASDAQ: ACHL) — that have been granted ODD status or may seek ODD status as they work to develop treatments for rare diseases.

Key Points: 
  • Four decades after the Orphan Drug Act was passed, hundreds of 'orphan' drugs have been approved for use.
  • Genprex's REQORSA Immunogene Therapy has gained both Orphan Drug Designation and Fast Track Designation.
  • Specifically, the report noted that 6,340 orphan drug designations were granted, representing drug development for 1,079 rare diseases.
  • For companies looking to help those in the underserved rare-disease space, an Orphan Drug Designation is the ideal pathway to progress.

Pathways to Progress: How Orphan Drug Designation Sparks Biotech Transformation

Retrieved on: 
Wednesday, September 20, 2023
Atezolizumab, Diabetes, Neoplasm, Doctor of Philosophy, ODA, National library, Patient, GC, Disease, Prostate cancer, Carcinoma, CD47, Hope, Research, ACHL, Lung cancer, NLM, Orphan drug, Death, Drug development, Diagnosis, Growth, Rare, FDA, Legislation, Civil service commission, ODD, Roche, Orphan Drug Act of 1983, PharmaMar, Cancer, NSCLC, Treatment of lung cancer, ES, Therapy, Stomach, ORR, European Commission, File, United States National Library of Medicine, MedImmune, Inc. v. Genentech, Inc., Clinical trial, Woman, Lung, Fast track (FDA), SCLC, American Cancer Society, Achilles, Melanoma, OTCQX, Orphan, EC, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, FTD, RET, Food, Breast cancer, Pharmaceutical industry, Medical imaging, Genentech, Pembrolizumab

NEW YORK, Sept. 20, 2023 /PRNewswire/ -- More than 30 million people in the United States suffer with a rare disease; most of them are receiving no current treatment. Supporting the development and evaluation of new treatments, particularly for rare diseases, is a key priority for the U.S. Food & Drug Administration. As part of that process, the agency can grant Orphan Drug Designation (ODD) to a drug or biological product being developed to prevent, diagnose or treat a rare disease or condition. This designation is intended to spark innovation among biotech companies that are developing treatments for these patient populations, which by definition affect fewer than 200,000 people in the U.S., by providing incentives such as tax credits, user-fee exemptions and up to seven years of market exclusivity after FDA approval. Last month, Genprex Inc. (NASDAQ: GNPX) (Profile) was granted FDA orphan drug designation for its REQORSA(R) immunogene therapy in development for the treatment of small cell lung cancer (SCLC). The Phase 1/2 clinical trial, expected to dose the first patient in the fourth quarter of 2023, uses a combination of REQORSA and Genentech Inc.'s Tecentriq(R) as maintenance therapy in patients with extensive stage small cell lung cancer ("ES-SCLC") who did not develop tumor progression after receiving Tecentriq and chemotherapy as initial standard treatment. Genprex joins other companies — including Roche Holding AG ADR (OTCQX: RHHBY), Jazz Pharmaceuticals PLC (NASDAQ: JAZZ), ALX Oncology Holdings Inc. (NASDAQ: ALXO) and Achilles Therapeutics PLC (NASDAQ: ACHL) — that have been granted ODD status or may seek ODD status as they work to develop treatments for rare diseases.

Key Points: 
  • Four decades after the Orphan Drug Act was passed, hundreds of 'orphan' drugs have been approved for use.
  • Genprex's REQORSA Immunogene Therapy has gained both Orphan Drug Designation and Fast Track Designation.
  • Specifically, the report noted that 6,340 orphan drug designations were granted, representing drug development for 1,079 rare diseases.
  • For companies looking to help those in the underserved rare-disease space, an Orphan Drug Designation is the ideal pathway to progress.

PepGen Inc. Announces Clearance of CTA by Health Canada to Begin the FREEDOM-DM1 Phase 1 Study of PGN-EDODM1 in Patients with Myotonic Dystrophy Type 1

Retrieved on: 
Wednesday, September 6, 2023
Orphan Drug Act of 1983, Neuromuscular disease, PGN, Myotonic Dystrophy Foundation, NOL, NMD, DM1, Health Canada, CTA, SAD, Patient, FDA, Conference, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, MDF, IND, Food, Safety, D.C, Disease, Pharmaceutical industry, Biology

In addition, the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to PGN-EDODM1 for the treatment of myotonic dystrophy type 1.

Key Points: 
  • In addition, the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to PGN-EDODM1 for the treatment of myotonic dystrophy type 1.
  • The FREEDOM-DM1 clinical trial is a randomized, placebo-controlled, single ascending dose (SAD) study, intended to enroll approximately 24 adult patients with DM1 to evaluate the safety and tolerability of PGN-EDODM1.
  • We expect to report initial results from this study in 2024,” said James McArthur, Ph.D., President and CEO of PepGen.
  • PepGen continues to work closely with the FDA to resolve the clinical hold in the United States as quickly as feasible.