Orphan Drug Act of 1983

AstraZeneca urges re-examination of unintended consequences of Inflation Reduction Act on American cancer and rare disease patients

Retrieved on: 
Friday, August 25, 2023
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To help protect access to medicines for cancer and rare disease patients, AstraZeneca today has filed a legal challenge to critical aspects of the drug price negotiation provisions of the Inflation Reduction Act (IRA).

Key Points: 
  • To help protect access to medicines for cancer and rare disease patients, AstraZeneca today has filed a legal challenge to critical aspects of the drug price negotiation provisions of the Inflation Reduction Act (IRA).
  • Dave Fredrickson, Executive Vice President, Oncology Business Unit, AstraZeneca, said: “Rare disease and cancer patients depend upon high-risk, low-probability drug development that takes many years to develop and aims for cure.
  • One example is LYNPARZA® (olaparib), a small-molecule cancer medicine approved in 2014 in the US for a small group of late-line ovarian cancer patients.
  • Additional trials added small groups of breast and pancreatic cancer patients, with the most recent indication in prostate cancer approved just this year – nine years later.

Wugen Begins First-in-Human Phase 1 Clinical Trial of WU-NK-101, its Lead Allogeneic Memory Natural Killer (NK) Cell Therapy for Patients with Relapsed or Refractory (R/R) Acute Myeloid Leukemia (AML)

Retrieved on: 
Tuesday, August 15, 2023
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Additionally, Wugen announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to WU-NK-101 for the treatment of AML.

Key Points: 
  • Additionally, Wugen announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to WU-NK-101 for the treatment of AML.
  • WU-NK-101 is Wugen’s lead memory natural killer (NK) cell therapy product and is comprised of cells optimized for anti-cancer function.
  • WU-NK-101 is produced with the company’s proprietary MonetaTM manufacturing platform, which enables robust generation of off-the-shelf memory NK cell-based therapies.
  • “Early clinical studies with memory NK cells demonstrated impressive response rates in patients with AML, even those with a high disease burden.

Propanc Biopharma’s Clinical Candidate PRP Targeting Pancreatic Cancer Market Forecast to Reach $6.93 Billion by 2030

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Tuesday, July 25, 2023
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Propanc Biopharma, Inc. (OTC Pink: PPCB) (“Propanc” or the “Company”), a biopharmaceutical company developing novel cancer treatments for patients suffering from recurring and metastatic cancer, today announced that the Company’s clinical candidate PRP targeting the pancreatic cancer market is forecast to reach $6.93 Billion by 2030, according to a report published by The Brainy Insights, a market research company.

Key Points: 
  • Propanc Biopharma, Inc. (OTC Pink: PPCB) (“Propanc” or the “Company”), a biopharmaceutical company developing novel cancer treatments for patients suffering from recurring and metastatic cancer, today announced that the Company’s clinical candidate PRP targeting the pancreatic cancer market is forecast to reach $6.93 Billion by 2030, according to a report published by The Brainy Insights, a market research company.
  • According to the report, the global pancreatic cancer market is expected to grow at a compound annual growth rate (CAGR) of 8.13% during the forecast period between 2022 to 2030.
  • As a result of laboratory and clinical studies undertaken in pancreatic cancer, Propanc applied for and received Orphan Drug Designation from the US FDA for the use of PRP to treat pancreatic cancer.
  • Orphan Drug Designation status of PRP has been granted from the US Food and Drug Administration (FDA) for treatment of pancreatic cancer.

Orphan Drugs and Rare Diseases Conference: Life-saving Therapies, Critical Issues, and Strategies to Enhance Orphan Drug Development in near future (London, United Kingdom - October 9-10, 2023) - ResearchAndMarkets.com

Retrieved on: 
Friday, July 7, 2023
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The 4th Annual Orphan Drugs and Rare Diseases Conference, to be held on October 9-10 in London, UK, aims to provide a valuable and broad platform to discuss recent advances in the development of life-saving therapies, critical issues, and strategies to enhance orphan drug development in near future.

Key Points: 
  • The 4th Annual Orphan Drugs and Rare Diseases Conference, to be held on October 9-10 in London, UK, aims to provide a valuable and broad platform to discuss recent advances in the development of life-saving therapies, critical issues, and strategies to enhance orphan drug development in near future.
  • With the enforcement of the 1983 Orphan Drug Act (ODA), manufacturers are obliged to scale production to treat rare/orphan diseases, leading to a drastic advancement in the landscape of orphan drugs.
  • The present-day challenges, including constrained resources, disease severity, limited patient access, and a lack of available knowledge in rare diseases, necessitate the discovery of innovative solutions to hasten orphan drug development.
  • Participate in discussions with experts on how to tackle the critical issues shaping the future of orphan drugs.

Final Inflation Reduction Act Guidance Risks Stifling Innovation for Rare Disease Community

Retrieved on: 
Friday, June 30, 2023
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Unfortunately, today's guidance threatens vital innovation for the thousands of rare diseases that lack FDA-approved therapies.

Key Points: 
  • Unfortunately, today's guidance threatens vital innovation for the thousands of rare diseases that lack FDA-approved therapies.
  • "History tells us without the right incentives, rare disease therapies are not developed, which is why the Orphan Drug Act was created 40 years ago," adds Saltonstall.
  • We will also continue to work with the Biden Administration to ensure the Inflation Reduction Act can best support people living with a rare disease," commits Saltonstall.
  • While certain aspects of today's guidance are deeply disappointing, NORD is committed to ensuring the Inflation Reduction Act helps meet the complex needs of the more than 25 million Americans living with a rare disease.

Global Rare Disease Clinical Trials Market Report 2023: Sector is Expected to Reach $24.25 Billion by 2030 at a CAGR of 9.7% - ResearchAndMarkets.com

Retrieved on: 
Wednesday, May 31, 2023
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Government initiatives that encourage product development, such as the Orphan Drug Act, are further expected to assist industry growth.

Key Points: 
  • Government initiatives that encourage product development, such as the Orphan Drug Act, are further expected to assist industry growth.
  • According to the National Institutes of Health (NIH), there are more than 7,000 rare diseases worldwide.
  • The growing awareness among people regarding rare diseases and growing interest in developing potential treatments for treating rare diseases are expected to support market growth.
  • For instance, in February 2022, the Health Research Board funded USD 1.1 million to support rare disease clinical trials.

Theravance Biopharma, Inc. Announces Orphan Drug Designation Granted to Ampreloxetine for the Treatment of Symptomatic Neurogenic Orthostatic Hypotension in Patients with Multiple System Atrophy

Retrieved on: 
Tuesday, May 9, 2023
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DUBLIN, May 9, 2023 /PRNewswire/ -- Theravance Biopharma, Inc. ("Theravance Biopharma" or the "Company") (NASDAQ: TBPH) today announced that the US Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) status to ampreloxetine for the treatment of symptomatic neurogenic orthostatic hypotension (nOH) in patients with multiple system atrophy (MSA).

Key Points: 
  • DUBLIN, May 9, 2023 /PRNewswire/ -- Theravance Biopharma, Inc. ("Theravance Biopharma" or the "Company") (NASDAQ: TBPH) today announced that the US Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) status to ampreloxetine for the treatment of symptomatic neurogenic orthostatic hypotension (nOH) in patients with multiple system atrophy (MSA).
  • MSA is a rare neurodegenerative disorder in which patients suffer autonomic dysfunction affecting movement, balance, heart rate and blood pressure.
  • "We are very pleased to have received Orphan Drug status for ampreloxetine in the treatment of nOH in patients with MSA," said Rick E Winningham, Chief Executive Officer.
  • "Ampreloxetine has the potential to improve MSA patients' and their caregivers' quality of life significantly.

Orphan Drugs Market is estimated to be US$ 869.6 billion by 2030 with a CAGR of 10.4% during the forecast period

Retrieved on: 
Tuesday, May 2, 2023
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Increasing prevalence of rare diseases: The prevalence of rare diseases is increasing, leading to a growing need for treatments for these conditions.

Key Points: 
  • Increasing prevalence of rare diseases: The prevalence of rare diseases is increasing, leading to a growing need for treatments for these conditions.
  • Competition from off-label drugs: Patients with rare diseases may turn to off-label use of existing drugs, which can compete with newly developed orphan drugs and reduce the market potential for these products.
  • The orphan drugs market is a global market, with a significant presence in North America, Europe, and Asia-Pacific.
  • North America is the largest market for orphan drugs, driven by factors such as favorable regulatory environment, high healthcare spending, and increasing prevalence of rare diseases.

XORTX Announces Receipt of FDA Orphan Drug Designation to Treat Autosomal Dominant Kidney Disease

Retrieved on: 
Friday, April 21, 2023
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CALGARY, Alberta, April 21, 2023 (GLOBE NEWSWIRE) -- XORTX Therapeutics Inc. ("XORTX" or the “Company”) (NASDAQ: XRTX | TSXV: XRTX | Frankfurt: ANU), a late-stage clinical pharmaceutical company focused on developing innovative therapies to treat progressive kidney disease, is pleased to announce the grant of Orphan Drug Designation for oxypurinol – “orphan-drug designation request of oxypurinol is granted for treatment of autosomal dominant polycystic kidney disease”.

Key Points: 
  • CALGARY, Alberta, April 21, 2023 (GLOBE NEWSWIRE) -- XORTX Therapeutics Inc. ("XORTX" or the “Company”) (NASDAQ: XRTX | TSXV: XRTX | Frankfurt: ANU), a late-stage clinical pharmaceutical company focused on developing innovative therapies to treat progressive kidney disease, is pleased to announce the grant of Orphan Drug Designation for oxypurinol – “orphan-drug designation request of oxypurinol is granted for treatment of autosomal dominant polycystic kidney disease”.
  • The orphan drug designation process (“ODD”) begins with an application to the US Food and Drug Administration (“FDA”) Orphan Drug Designation office, and is accompanied by a comprehensive package that includes the basic science, analysis of addressable number of individuals with autosomal dominant polycystic kidney disease (“ADPKD”) and description of the patients projected to be helped by a therapy.
  • Dr. Allen Davidoff stated, “Orphan drug designation represents a major milestone for the Company in pursuit of marketing approval for XORLOTM, our proprietary formulation of oxypurinol, and the XRx-008 program for ADPKD.
  • A tax credit of 25% of the qualified clinical drug testing costs awarded upon drug approval is also possible.

Ocean Biomedical (NASDAQ: OCEA) Will Target Pulmonary Fibrosis Discoveries as Novel Treatments of Hermansky-Pudlak Syndrome

Retrieved on: 
Thursday, April 6, 2023
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Providence, RI, April 06, 2023 (GLOBE NEWSWIRE) -- Ocean Biomedical, Inc. (NASDAQ: OCEA) announced today a strong commitment to targeting its pulmonary fibrosis treatment candidate, “OCF-203,” as a novel therapeutic for fatal pulmonary fibrotic conditions caused by Hermansky-Pudlak Syndrome (HPS). Discoveries in recent years by Ocean Biomedical’s Scientific Co-founder, Dr. Jack A. Elias, and his colleagues at Brown University, have revealed a new target and a new pathway for treating pulmonary tissue damage in Idiopathic Pulmonary Fibrosis (IPF). Experiments have also been performed with genetically modified “pale-ear” mice, which have the same mutations that are seen in patients with Hermansky-Pudlak Syndrome (HPS). These experiments demonstrated that the same small molecule may be effective in treating pulmonary fibrosis conditions in patients with HPS, especially the most deadly forms of that disease, HPS-1 and HPS-4.

Key Points: 
  • On Hermansky-Pudlak Syndrome (HPS) Awareness Day, Ocean Biomedical announces
    Providence, RI, April 06, 2023 (GLOBE NEWSWIRE) -- Ocean Biomedical, Inc. (NASDAQ: OCEA) announced today a strong commitment to targeting its pulmonary fibrosis treatment candidate, “OCF-203,” as a novel therapeutic for fatal pulmonary fibrotic conditions caused by Hermansky-Pudlak Syndrome (HPS).
  • Discoveries in recent years by Ocean Biomedical’s Scientific Co-founder, Dr. Jack A. Elias, and his colleagues at Brown University, have revealed a new target and a new pathway for treating pulmonary tissue damage in Idiopathic Pulmonary Fibrosis (IPF).
  • In four pulmonary fibrosis animal models, Ocean Biomedical’s OCF-203 has shown an 85% – 90% reduction in collagen accumulation.
  • In HPS-1 and HPS-4 patients pulmonary fibrosis occurs early in life (30s and 40s) and symptoms are often severe.