Posts in category: Rare Disease News

Longeveron Inc. (NASDAQ: LGVN) (“Longeveron” or “Company”), a clinical stage biotechnology company developing cellular therapies for chronic aging-related and certain life-threatening conditions, announced today that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) for Lomecel-B for the treatment of Hypoplastic Left Heart Syndrome (HLHS), a rare and life-threatening congenital heart defect in infants.

ODD is intended to assist and encourage companies to develop safe and effective therapies for the treatment of rare diseases or conditions. ODD positions Longeveron to be able to potentially leverage a range of financial and regulatory benefits, including government grants for conducting clinical trials, waiver of FDA user fees for the potential submission of a marketing application, and certain tax credits. Receiving ODD may also result in the product receiving seven years market exclusivity upon approval for use in the rare disease or condition for which the product was designated if all of the statutory and regulatory requirements are met.

“Adding to the Rare Pediatric Disease (RPD) designation already granted to Lomecel-B for treatment of HLHS, the FDA’s decision to grant ODD to Lomecel-B for this indication indicates the ongoing and unmet need for new therapies to treat infants with HLHS,” said Geoff Green, Chief Executive Officer at Longeveron. “Building on results from our completed Phase 1 safety-focused trial, we believe Lomecel-B has potential to improve outcomes for these severely impacted infants by way of repairing cardiac tissue and improving ventricular function. The combination of both RPD and ODD allows us to potentially move more efficiently through clinical development and regulatory review, and Lomecel-B may be eligible for a period of marketing exclusivity upon approval for this indication.”

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Disclaimer: BioPharma Global is not responsible for, and expressly disclaims all liability for, damages of any kind arising out of use, reference to, or reliance on any information contained within the article. Content available through the site may contain links and information to other websites. Links from BioPharma Global to third-party sites do not constitute an endorsement by BioPharma Global of the mentioned parties.

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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Soligenix (Nasdaq: SNGX) (Soligenix or the Company), a late-stage biopharmaceutical company focused on developing and commercializing products to treat rare diseases where there is an unmet medical need, announced today that it has been granted a Pediatric Investigation Plan (PIP) product-specific waiver from the Medicines and Healthcare products Regulatory Agency (MHRA) for HyBryte™ (SGX301 or synthetic hypericin), which has successfully concluded a Phase 3 pivotal clinical study for the treatment of early stage cutaneous T-cell lymphoma (CTCL).

The waiver was provided for all subsets of the pediatric population from birth to less than 18 years of age on the grounds that clinical studies in this rare population are not feasible. Earlier this year the European Medicines Agency (EMA) also granted a waiver to the Pediatric Investigational Plan requirements for the European Union (EU). With the withdrawal of the United Kingdom (UK) from the EU effective January 1, 2021, the MHRA became the UK’s standalone medicines and medical devices regulator.

“This achievement is an important regulatory milestone as we move forward with marketing applications worldwide,” stated Christopher J. Schaber, PhD, President and Chief Executive Officer of Soligenix. “The PIP waiver allows us to work towards advancing a marketing authorization application (MAA) in the UK in a more cost-effective manner since we will not need to expend resources to conduct a pediatric clinical study.”

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Disclaimer: BioPharma Global is not responsible for, and expressly disclaims all liability for, damages of any kind arising out of use, reference to, or reliance on any information contained within the article. Content available through the site may contain links and information to other websites. Links from BioPharma Global to third-party sites do not constitute an endorsement by BioPharma Global of the mentioned parties.

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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Reata Pharmaceuticals, Inc. (Nasdaq: RETA), (“Reata,” the “Company,” “our,” “us,” or “we”), a clinical-stage biopharmaceutical company, today announced the U.S. Food and Drug Administration (“FDA”) has granted Fast Track Designation for omaveloxolone for the treatment of Friedreich’s ataxia.

“We are pleased to receive Fast Track Designation as it highlights the potential of omaveloxolone to address a significant unmet medical need for the treatment of patients with Friedreich’s ataxia, a severe and devastating disease,” said Warren Huff, Reata’s President and Chief Executive Officer. “We remain committed to submitting our New Drug Application during the first quarter of 2022 and continue working with the FDA to secure regulatory approval as quickly as possible.”

The Fast Track program is designed to accelerate the development and review of products such as omaveloxolone, which are intended to treat serious diseases and for which there is an unmet medical need. Fast Track Designation enables more frequent communication with the FDA and eligibility for FDA programs such as priority review and rolling review, if relevant criteria are met.

About Friedreich’s Ataxia

Friedreich’s ataxia is a rare, genetic, life-shortening, debilitating, and degenerative neuromuscular disorder, which is normally diagnosed during adolescence. Friedreich’s ataxia is caused by a trinucleotide repeat expansion in the first intron of the frataxin gene, which encodes the mitochondrial protein frataxin. Pathogenic repeat expansions can lead to impaired transcription and reduced frataxin expression, which can result in mitochondrial iron overload and poor cellular iron regulation, increased sensitivity to oxidative stress, and impaired mitochondrial ATP production. Patients with Friedreich’s ataxia experience symptoms in childhood, including progressive loss of coordination, muscle weakness, and fatigue that commonly results in motor incapacitation with patients requiring a wheelchair in their teens or early 20s. Patients with Friedreich’s ataxia may also experience visual impairment, hearing loss, diabetes, and cardiomyopathy. Based on literature and proprietary research, we believe Friedreich’s ataxia affects approximately 5,000 children and adults in the United States and 22,000 individuals globally. There are currently no approved therapies for the treatment of patients with Friedreich’s ataxia.

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Disclaimer: BioPharma Global is not responsible for, and expressly disclaims all liability for, damages of any kind arising out of use, reference to, or reliance on any information contained within the article. Content available through the site may contain links and information to other websites. Links from BioPharma Global to third-party sites do not constitute an endorsement by BioPharma Global of the mentioned parties.

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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CRISPR Therapeutics (Nasdaq: CRSP), a biopharmaceutical company focused on creating transformative gene-based medicines for serious diseases, today announced that the U.S. Food and Drug Administration (FDA) granted Regenerative Medicine Advanced Therapy (RMAT) designation to CTX110™, its wholly-owned allogeneic CAR-T cell therapy targeting CD19+ B-cell malignancies.

“This RMAT designation is based on the encouraging clinical data we have presented thus far, and it is an important milestone that recognizes the transformative potential of CTX110 for the treatment of hematological malignancies,” said Samarth Kulkarni, Ph.D., Chief Executive Officer of CRISPR Therapeutics. “We look forward to working closely with the FDA as we continue our efforts to bring this important new therapeutic modality to patients.”

Established under the 21st Century Cures Act, RMAT designation is a dedicated program designed to expedite the drug development and review processes for promising pipeline products, including genetic therapies. A regenerative medicine therapy is eligible for RMAT designation if it is intended to treat, modify, reverse or cure a serious or life-threatening disease or condition, and preliminary clinical evidence indicates that the drug or therapy has the potential to address unmet medical needs for such disease or condition. Similar to Breakthrough Therapy designation, RMAT designation provides the benefits of intensive FDA guidance on efficient drug development, including the ability for early interactions with FDA to discuss surrogate or intermediate endpoints, potential ways to support accelerated approval and satisfy post-approval requirements, potential priority review of the biologics license application (BLA) and other opportunities to expedite development and review.

To Read the Complete Article at BioSpace News Click Here

Disclaimer: BioPharma Global is not responsible for, and expressly disclaims all liability for, damages of any kind arising out of use, reference to, or reliance on any information contained within the article. Content available through the site may contain links and information to other websites. Links from BioPharma Global to third-party sites do not constitute an endorsement by BioPharma Global of the mentioned parties.

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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Mirati Therapeutics (NASDAQ: MRTX), a clinical-stage targeted oncology company, announced today that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation to adagrasib for the potential treatment of patients with non-small cell lung cancer (NSCLC) who harbor the KRAS^G12C mutation following prior systemic therapy.

Breakthrough Therapy Designation is an FDA program designed to expedite the development and regulatory review of drugs that have demonstrated preliminary clinical evidence of a substantial improvement over available therapy in the treatment of patients with serious diseases on at least one clinically significant endpoint.

The designation for adagrasib is supported by preliminary results from the registrational Phase 1/2 KRYSTAL-01 trial in patients with advanced NSCLC, whose cancer had progressed following prior treatment with immunotherapy and/or chemotherapy.

“We are pleased to receive this breakthrough therapy designation for adagrasib, which emphasizes the significant need for new treatment options for patients with lung cancer who harbor the KRAS^G12C mutation,” said Charles M. Baum, M.D., Ph.D., president and chief executive officer, Mirati Therapeutics, Inc. “We look forward to submitting a New Drug Application for adagrasib in the second half of this year and further advancing adagrasib across a broad development plan with the goal of improving clinical outcomes in patients with KRAS^G12C mutated cancers.”

To Read the Complete Article at BioSpace News Click Here

Disclaimer: BioPharma Global is not responsible for, and expressly disclaims all liability for, damages of any kind arising out of use, reference to, or reliance on any information contained within the article. Content available through the site may contain links and information to other websites. Links from BioPharma Global to third-party sites do not constitute an endorsement by BioPharma Global of the mentioned parties.

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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BeiGene Co.,Ltd (NASDAQ: BGNE; HKEX: 06160) announced today that the European Commission (EC) approved BRUKINSA^® (zanubrutinib) for the treatment of adult patients with Waldenström’s macroglobulinemia (WM) who have received at least one prior therapy or for the first-line treatment of patients unsuitable for chemo-immunotherapy. The approval is applicable to all 27 European Union (EU) member states, plus Iceland and Norway. BeiGene is working to make this new treatment option available to WM patients in the EU as quickly as possible.

“BTK inhibition is an established mode of treatment for patients with WM, and the approval of BRUKINSA provides an important new option for patients with WM that may offer improved outcomes,” said Prof. Christian Buske, Medical Director at the University Hospital Ulm, Germany, and a trial investigator of the ASPEN study. “Patients and their physicians in the EU will soon have access to an innovative medicine that has potential to offer deep and durable responses and improved tolerability, as seen in the ASPEN trial.”

The EC approval for BRUKINSA follows a positive opinion granted in September by the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA), based on the results of the ASPEN trial. Although the primary endpoint of statistical superiority related to deep response, very good partial response (VGPR) or better, was not met, BRUKINSA demonstrated clinical benefit with advantages in safety compared to ibrutinib.¹ Read more about the positive CHMP opinion and ASPEN trial results here.

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Disclaimer: BioPharma Global is not responsible for, and expressly disclaims all liability for, damages of any kind arising out of use, reference to, or reliance on any information contained within the article. Content available through the site may contain links and information to other websites. Links from BioPharma Global to third-party sites do not constitute an endorsement by BioPharma Global of the mentioned parties.

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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REGENXBIO Inc. (Nasdaq: RGNX) today announced the U.S. Food and Drug Administration (FDA) granted Orphan Drug Designation for RGX-202, a potential one-time gene therapy for the treatment of Duchenne muscular dystrophy (Duchenne). RGX-202 is designed to deliver a novel, optimized microdystrophin transgene with a unique C-terminal domain and a muscle specific promoter to support targeted therapy for improved resistance to muscle damage associated with Duchenne. RGX-202 uses REGENXBIO’s proprietary NAV^® AAV8 vector. 

“This important designation is a milestone in the development of RGX-202 and highlights the need for potential new treatment options for patients with Duchenne,” said Olivier Danos, Ph.D., Chief Scientific Officer of REGENXBIO. “The novel microdystrophin transgene in RGX-202 includes coding regions that retain essential functional elements of naturally occurring dystrophin to potentially improve muscle strength and resistance in patients with Duchenne. We look forward to advancing this one-time gene therapy into the clinic.” 

REGENXBIO expects to submit an Investigational New Drug (IND) application to the FDA for RGX-202 by the end of 2021. Commercial-scale cGMP material has already been produced at 1,000 liter capacity using REGENXBIO’s suspension cell culture manufacturing process, and the Company’s internal cGMP facility is expected to allow for production up to 2,000 liters for the clinical development of RGX-202.

To Read the Complete Article at BioSpace News Click Here

Disclaimer: BioPharma Global is not responsible for, and expressly disclaims all liability for, damages of any kind arising out of use, reference to, or reliance on any information contained within the article. Content available through the site may contain links and information to other websites. Links from BioPharma Global to third-party sites do not constitute an endorsement by BioPharma Global of the mentioned parties.

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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The U.S. Food and Drug Administration (FDA) has given fast track designation to Prilenia Therapeutics’ pridopidine as a potential oral treatment for Huntington’s disease.

This designation is given to therapies that show considerable potential in addressing serious conditions for which available treatments fall short. It is meant to speed their clinical development and regulatory review — enabling a rolling review and qualification for accelerated approval and priority review.

Rolling review means the company can submit sections of the regulatory application for review as they come ready, rather than waiting for every section to be completed before the application can be reviewed by the agency.

“Receipt of Fast Track designation from the FDA underscores the urgency to address a significant unmet need for patients with Huntington’s Disease,” Michael R. Hayden, PhD, Prilenia’s CEO and founder, said in a press release.

Huntington’s “is one of the most devastating neurodegenerative disorders, impacting not only patients but their families,” Hayden said, adding that “at the present time, there is no approved treatment to delay onset or slow disease progression.”

To Read the Complete Article at Huntington’s Disease News Click Here

Disclaimer: BioPharma Global is not responsible for, and expressly disclaims all liability for, damages of any kind arising out of use, reference to, or reliance on any information contained within the article. Content available through the site may contain links and information to other websites. Links from BioPharma Global to third-party sites do not constitute an endorsement by BioPharma Global of the mentioned parties.

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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Ascentage Pharma (6855.HK), a global biopharmaceutical company engaged in developing novel therapies for cancers, chronic hepatitis B (CHB), and age-related diseases, today announced that the European Commission (EC) has granted the company’s novel drug candidate, olverembatinib (HQP1351), an Orphan Designation for the treatment of chronic myeloid leukemia (CML). This is the first Orphan Designation granted to Ascentage Pharma’s drug candidates in the European Union (EU), and the second granted to olverembatinib globally, following the designation by the US Food and Drug Administration (FDA).

The term “orphan medicines” refers to pharmaceutical products developed for the prevention, diagnosis, and treatment of rare diseases or conditions. In the EU, Orphan Designations are granted by the EC based on the opinions of the Committee for Orphan Medicinal Products (COMP) of the European Medicines Agency (EMA), and only those therapies treating life-threatening or chronically debilitating diseases or conditions affecting less than 5 in 10,000 people in the EU and represent huge unmet medical needs are granted the Orphan Designation. Moreover, designated drugs must be able to demonstrate through non-clinical and clinical data that it can potentially provide greater therapeutic benefit than existing therapies. The EMA offers a range of incentives to encourage the development of designated orphan medicines. This Orphan Designation for olverembatinib qualifies the drug candidate for great regulatory supports in the subsequent clinical development and commercialization in the European Union, including protocol assistance, fee reductions, and most importantly, 10 years of market exclusivity upon approval.

CML is a rare hematologic malignancy that has an annual incidence of 2.43 per 10,000^[1] in the 27 member nations of the EU. BCR-ABL tyrosine kinase inhibitors (TKIs) have significantly improved the clinical management of CML. However, despite clinical benefits offered by the first- and second-generation TKIs, many patients develop drug resistance. Such acquired resistance to TKIs is a major challenge in the treatment of CML. BCR-ABL kinase mutations represent a key mechanism of acquired drug resistance; T315I, which is the most- common drug-resistant mutation, occurs in about 25% of patients with drug-resistant CML. Patients with T315I-mutant CML are resistant to both first- and second-generation BCR-ABL inhibitors, hence presenting an urgent and high unmet medical need for third-generation BCR-ABL inhibitors to more effectively target the T315I mutation.

To Read the Complete Article at Pharmacy Times Click Here

Disclaimer: BioPharma Global is not responsible for, and expressly disclaims all liability for, damages of any kind arising out of use, reference to, or reliance on any information contained within the article. Content available through the site may contain links and information to other websites. Links from BioPharma Global to third-party sites do not constitute an endorsement by BioPharma Global of the mentioned parties.

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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CRISPR Therapeutics (Nasdaq: CRSP), a biopharmaceutical company focused on creating transformative gene-based medicines for serious diseases, today announced that the U.S. Food and Drug Administration (FDA) granted Regenerative Medicine Advanced Therapy (RMAT) designation to CTX110™, its wholly-owned allogeneic CAR-T cell therapy targeting CD19+ B-cell malignancies.

“This RMAT designation is based on the encouraging clinical data we have presented thus far, and it is an important milestone that recognizes the transformative potential of CTX110 for the treatment of hematological malignancies,” said Samarth Kulkarni, Ph.D., Chief Executive Officer of CRISPR Therapeutics. “We look forward to working closely with the FDA as we continue our efforts to bring this important new therapeutic modality to patients.”

Established under the 21st Century Cures Act, RMAT designation is a dedicated program designed to expedite the drug development and review processes for promising pipeline products, including genetic therapies. A regenerative medicine therapy is eligible for RMAT designation if it is intended to treat, modify, reverse or cure a serious or life-threatening disease or condition, and preliminary clinical evidence indicates that the drug or therapy has the potential to address unmet medical needs for such disease or condition. Similar to Breakthrough Therapy designation, RMAT designation provides the benefits of intensive FDA guidance on efficient drug development, including the ability for early interactions with FDA to discuss surrogate or intermediate endpoints, potential ways to support accelerated approval and satisfy post-approval requirements, potential priority review of the biologics license application (BLA) and other opportunities to expedite development and review.

To Read the Complete Article at Pharmacy Times Click Here

Disclaimer: BioPharma Global is not responsible for, and expressly disclaims all liability for, damages of any kind arising out of use, reference to, or reliance on any information contained within the article. Content available through the site may contain links and information to other websites. Links from BioPharma Global to third-party sites do not constitute an endorsement by BioPharma Global of the mentioned parties.

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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