orphan drugs Archives - Page 5 of 23

Tezepelumab granted Orphan Drug Designation in the US for eosinophilic esophagitis

October 8, 2021

by administrator Drug Discovery News Rare Disease Highlight Rare Disease News Regulatory News

Tezepelumab has been granted Orphan Drug Designation (ODD) in the US by the Food and Drug Administration (FDA) for the treatment of eosinophilic esophagitis (EoE).

Tezepelumab is being developed by AstraZeneca in collaboration with Amgen and is under Priority Review for patients with asthma in the US. The FDA grants ODD status to medicines and potential new medicines intended for the treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 people in the US.

EoE is a rare, chronic, inflammatory disease that occurs when eosinophils, a type of white blood cell, accumulate in the esophagus. In addition to eosinophils, other cells including mast cells, T-cells and fibroblasts drive injury, inflammation and detrimental tissue remodelling. If the disease is not effectively treated it can make eating difficult or uncomfortable, potentially leading to chronic pain, difficulty swallowing, poor growth, malnutrition and weight loss.

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BioPharma Global is a mission-driven corporation, operating like a not-for-profit, dedicated to using our FDA and EMA regulatory expertise and knowledge of various therapeutic areas to help drug developers advance treatments for the disease communities with a high unmet medical need. If you are a drug developer seeking regulatory support for Orphan Drug designation, Fast Track designation, Breakthrough Therapy designation, other FDA/EMA expedited programs, type A, B (pre-IND, EOPs), or C meeting assistance, or IND filings, the BioPharma Global team can help. Contact us today to arrange a 30-minute introductory call.

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NuCana Receives Fast Track Designation from the U.S. Food and Drug Administration for Acelarin® (NUC-1031) for the Treatment of Biliary Tract Cancer

October 7, 2021

by administrator Drug Discovery News Rare Disease Highlight Rare Disease News Regulatory News

NuCana plc (NASDAQ: NCNA) announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to Acelarin (NUC-1031), currently being evaluated in a Phase III study (NuTide:121) for the first-line treatment of patients with advanced biliary tract cancer. Fast Track is a process designed to facilitate the development and expedite the review of drugs to treat serious conditions and address an unmet medical need.

“We are very pleased that the FDA recognizes the potential of Acelarin to address the significant unmet need of patients with biliary tract cancer,” said Hugh S. Griffith, NuCana’s Founder and Chief Executive Officer. “We recently announced enrollment of 418 evaluable patients in our Phase III study, which is expected to enable the first interim analysis in the first half of 2022. This has the potential to allow for an accelerated approval of a new drug application (NDA) for Acelarin in the United States. With both Fast Track and Orphan Drug designations in place, we look forward to working closely with the FDA in our efforts to gain approval for Acelarin as the first approved front-line treatment option for patients with biliary tract cancer.”

About Fast Track Designation

Fast track is a process designed to facilitate the development, and expedite the review of drugs to treat serious conditions and fill an unmet medical need. The purpose is to get important new drugs to the patient earlier. Filling an unmet medical need is defined as providing a therapy where none exists or providing a therapy which may be potentially better than available therapy. Once a drug receives Fast Track designation, early and frequent communication between the FDA and a drug company is encouraged throughout the entire drug development and review process. The frequency of communication assures that questions and issues are resolved quickly, often leading to earlier drug approval and access by patients.

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FDA Grants Orphan Drug Designation (ODD) to AlloVir’s Posoleucel, an Allogeneic, Off-the-Shelf, Multi-Virus Specific T-Cell Therapy, for the Treatment of Virus-Associated Hemorrhagic Cystitis

October 6, 2021

by administrator Drug Discovery News Rare Disease Highlight Rare Disease News Regulatory News

AlloVir, Inc. (Nasdaq: ALVR), a late clinical-stage cell therapy company, today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to posoleucel (Viralym-M, ALVR105) for the treatment of virus-associated hemorrhagic cystitis (HC). Posoleucel is an investigational, allogeneic, off-the-shelf, multi-virus specific T cell therapy under development for the treatment and prevention of serious diseases caused by six devastating viral pathogens: BK virus (BKV) and the related polyomavirus JC virus (JCV), cytomegalovirus (CMV), human herpes virus-6 (HHV-6), Epstein Barr virus (EBV) and adenovirus (AdV).

An inflammatory disease of the bladder, virus-associated HC is a serious complication of hematopoietic stem cell transplantation (HSCT) that can significantly prolong hospitalization and increase mortality and for which there are currently no approved or effective antiviral treatment options. The company’s Phase 3 study of posoleucel for the treatment of virus-associated HC is ongoing and enrolling adult and pediatric patients following allogeneic HSCT (allo-HSCT).

“This Orphan Drug Designation acknowledges the urgent need for new treatment options for patients who have undergone hematopoietic stem cell transplantation and are at risk for developing viral infections and hemorrhagic cystitis,” said Ercem Atillasoy, M.D., Chief Regulatory and Safety Officer, AlloVir. “We look forward to working with the FDA and regulators around the globe as we advance this therapy for patients in need.”

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Zai Lab Partner Turning Point Therapeutics Granted Breakthrough Therapy Designation for Repotrectinib Treatment in Patients With NTRK-Positive, TKI-Pretreated Advanced Solid Tumors

October 5, 2021

by administrator Drug Discovery News Rare Disease Highlight Rare Disease News Regulatory News

Zai Lab Limited (NASDAQ: ZLAB; HKEX: 9688) partner Turning Point Therapeutics, Inc. (NASDAQ: TPTX), a precision oncology company developing next-generation therapies that target genetic drivers of cancer, today announced the U.S. Food and Drug Administration (FDA) granted a seventh regulatory designation to lead drug candidate, repotrectinib.

Breakthrough Therapy designation was granted for the treatment of patients with advanced solid tumors that have an NTRK gene fusion who have progressed following treatment with one or two prior TRK tyrosine kinase inhibitors, with or without prior chemotherapy, and have no satisfactory alternative treatments. The company is planning to discuss next steps towards potential registration of repotrectinib in this patient population at a Type B meeting with the FDA anticipated in the first half of 2022.

Breakthrough Therapy designation is granted by the FDA to expedite the development and regulatory review of an investigational medicine that is intended to treat a serious or life-threatening condition. The criteria for Breakthrough Therapy designation require preliminary clinical evidence that demonstrates the drug may have substantial improvement on at least one clinically significant endpoint over available therapy.

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ImmixBio Announces FDA Orphan Drug Designation for IMX-110 for the Treatment of Soft Tissue Sarcoma

October 4, 2021

by administrator Drug Discovery News Rare Disease Highlight Rare Disease News Regulatory News

Immix Biopharma, Inc (“ImmixBio”), a biotechnology company pioneering Tissue Specific Therapeutics (TSTx)^TMfor oncology and inflammation, today announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation (ODD) to IMX-110 for the treatment of soft tissue sarcoma.

The FDA’s Office of Orphan Products Development grants orphan designation status to drugs and biologics that are intended for the safe and effective treatment, diagnosis or prevention of rare diseases, or conditions that affect fewer than 200,000 people in the U.S. Orphan Drug Designation provides certain benefits, including financial incentives, to support clinical development and the potential for up to 7 years of market exclusivity in the U.S. upon regulatory approval.

“We are pleased to receive FDA’s orphan drug designation in soft tissue sarcoma for IMX-110, the first clinical-stage product of our SMAR_xT Tissue-Specific Platform,” said Ilya Rachman MD PhD, ImmixBio Founder & CEO. “We are thrilled to potentially contribute to expanding therapeutic options for oncology patients, beginning with soft tissue sarcoma.”

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U.S. FDA Accepts Filings for Amicus’ AT-GAA for the Treatment of Pompe Disease

October 1, 2021

by administrator Drug Discovery News Rare Disease Highlight Rare Disease News Regulatory News

Amicus Therapeutics (Nasdaq: FOLD) today announced that the U.S. Food and Drug Administration (FDA) has accepted for review the Biologics License Application (BLA) for cipaglucosidase alfa and the New Drug Application (NDA) for miglustat for AT-GAA, the Company’s investigational two-component therapy for the treatment of Pompe disease. Pompe disease is a rare genetic disease that causes premature death and has a debilitating effect on people’s lives. The U.S. represents the single largest geography for Amicus to positively impact the lives of people with Pompe disease.

The FDA has set a Prescription Drug User Fee Act (PDUFA) action date of May 29, 2022 for the NDA and July 29, 2022 for the BLA. The BLA and NDA are based on the evaluation of the effects of AT-GAA in Pompe disease patients and its safety profile, which include data from the Phase 1/2 and Phase 3 PROPEL studies as well as data from the open-label extension study.

John F. Crowley, Chairman and Chief Executive Officer of Amicus Therapeutics Inc., stated, “The FDA’s acceptance of these filings is an immensely important step forward for people living with Pompe disease and their families in the United States. Patients need new medicines as soon as possible. We will work with great urgency with the FDA as they review the applications over the course of the coming months. In parallel, we are diligently working towards additional regulatory submissions outside of the U.S. With today’s announcement, we remain confident in the potential of this medicine to become the next standard of care in Pompe disease.”

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Mirum Snags First Drug Approval for Rare, Genetic Liver Disorder

September 30, 2021

by administrator Drug Discovery News Rare Disease Highlight Rare Disease News Regulatory News

Mirum Pharmaceuticals has received approval from the U.S. Food and Drug Administration (FDA) to start selling its oral medication LIVMARLI to treat cholestatic pruritus in patients diagnosed with Alagille syndrome (ALGS).

LIVMARLI (maralixibat) is indicated for ages one year and older. It is an orally administered, once-daily ileal bile acid transporter (IBAT) inhibitor. It will now be accessible to patients with a prescription through Mirum Access Plus, the company’s patient support services program and single-source specialty pharmacy. The MAP program works with healthcare providers and families to provide access support, insurance coverage, and financial support options for the drug.

ALGS is a rare genetic disease caused by bile duct abnormalities, leading to progressive liver disease. The shrunken or malformed bile ducts cause cholestasis, which eventually develops into inflammation and liver injury. It affects 2,000 to 2,5000 children in the U.S. alone. The estimated incidence of ALGS is one in every 30,000 people.

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Immunoforge Inc. receives U.S. FDA ODD for Treatment of Polymyositis

September 30, 2021

by administrator Drug Discovery News Rare Disease Highlight Rare Disease News Regulatory News

Immunoforge (Co-CEOs Sung-min Ahn, Kiho Chang), a company specializing in developing new drugs for rare musculoskeletal diseases, has reported that their new drug candidate PF1801 has received Orphan Drug Designation (ODD) from the U.S. FDA for the treatment of Polymyositis.

The FDA designates solutions for rare incurable diseases or life-threatening diseases as Orphan Drugs. With this Orphan Drug Designation, Immunoforge will receive various benefits such as exemption from NDA/BLA application fees, tax credits for qualified clinical trials, and exclusive rights for seven years upon marketing approval.

Polymyositis is a disease in which muscles weaken due to inflammatory reactions; the cause of the disease has not yet been identified. Immunoforge states that “This Orphan Drug Designation is of great significance in confirming the potential of PF1801 as a treatment for rare musculoskeletal diseases, and we hope that it will serve as a source of hope for patients suffering from rare diseases with the various new drugs we are developing.”

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FDA Grants Catalyst Biosciences Orphan Drug Designation for MarzAA for the Treatment of Factor VII Deficiency

September 29, 2021

by administrator Drug Discovery News Rare Disease Highlight Rare Disease News Regulatory News

Catalyst Biosciences, Inc. (NASDAQ: CBIO) today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Disease Designation (ODD) for its lead product candidate, subcutaneous Marzeptacog alfa (activated), or MarzAA, for the treatment of Factor VII Deficiency (FVIID). MarzAA was previously granted ODD and Fast Track Designation (FTD) for treatment of Hemophilia A/B with inhibitors and FTD for the treatment of FVIID.

“Receiving a second orphan drug designation in addition to two FTDs for MarzAA demonstrates its potential in treating multiple rare bleeding disorders” said Nassim Usman, Ph.D., president and chief executive officer of Catalyst.

The FDA grants Orphan Drug Designation to drugs and biologics intended for the safe and effective treatment, diagnosis or prevention of rare diseases or conditions affecting fewer than 200,000 people in the United States. Orphan Drug Designation provides benefits to drug developers designed to support the developments of drugs and biologics for small patient populations with unmet medical needs. These benefits include assistance in the drug development process, tax credits for clinical costs, exemptions from certain FDA fees and seven years of marketing exclusivity.

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Rare Disease Innovation and Data Sharing Accelerated by New RDCA-DAP Program

September 28, 2021

by administrator Drug Discovery News Rare Disease Highlight Rare Disease News Regulatory News

The Rare Disease Cures Accelerator-Data and Analytics Platform initiative (RDCA-DAP®) officially launched its next phase on Tuesday establishing itself as the leading platform to accelerate rare disease treatment innovation. The launch was part of an all-day virtual workshop and annual meeting of rare disease stakeholders, featuring a platform demonstration, industry expert presentations and attendance by over 400 patients, providers, researchers, clinicians, biopharmaceutical companies, regulatory reviewers and scientists.

RDCA-DAP is an FDA-funded initiative that will serve as a centralized and standardized base to host and share de-identified rare diseases data and to support treatment innovation. It was created through a partnership between Critical Path Institute (C-Path), the National Organization for Rare Disorders (NORD®) and the U.S. Food and Drug Administration (FDA) and has grown to include many others throughout the rare disease community.

“There is a lot of enthusiasm for seeing this platform become all it can be for patients and drug developers. It is not just about the data. This is a place to generate solutions in a highly collaborative way,” said Jeff Barrett, Ph.D., F.C.P., C-Path Senior Vice President and RDCA-DAP Lead. “We expect RDCA-DAP to be a place to promote candid dialogue, optimize patient selection and trial design, and co-develop the tools to further de-risk decision making for rare disease drug development.”

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