11-23-2021

Ascentage Pharma’s Olverembatinib Granted Orphan Designation by the European Commission for the Treatment of Chronic Myeloid Leukemia

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.

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11-22-2021

CRISPR Therapeutics (CRSP) Announces FDA RMAT Designation Granted to CTX110 for the Treatment of Relapsed or Refractory CD19+ B-cell malignancies

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.

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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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11-19-2021

U.S. FDA CLEARS BIOMARIN’S VOXZOGO AS FIRST APPROVED THERAPY FOR ACHONDROPLASIA

BioMarin Pharmaceutical scored a first on Friday and provided an option for patients with a rare disease for which there is little recourse. The U.S. Food and Drug Administration approved Voxzogo (vosoritide) to improve growth in children five years of age and older with achondroplasia, a rare genetic disorder that causes the most common form of dwarfism.

The drug, a once-daily injection, has been approved specifically for children who have open epiphyses, or growth plates, which gives them the potential for growth. For those children with achondroplasia, a mutation prevents the growth of that bone. Voxzogo, a C-type natriuretic peptide (CNP) analog, corrects that by acting as a positive regulator of the signaling pathway downstream of the fibroblast growth factor receptor 3 gene. With the FDA nod, Voxzogo becomes the first drug approved in the U.S to treat the condition. Prior to the approval, there were some drastic surgical options available to patients, but no therapeutics.

Achondroplasia is a genetic condition that causes severely short stature and disproportionate growth. The average height of an adult with achondroplasia is approximately four feet.

Voxzogo was greenlit under accelerated approval based on clinical data demonstrating an improvement in annualized growth velocity (AGV). Under the framework of accelerated approval, California-based BioMarin will be required to continue to provide follow-up data to the FDA. The company said it intends to use ongoing open-label extension studies compared to available natural history to fulfill the requirement.

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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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11-18-2021

BerGenBio Receives FDA Fast Track Designation For Bemcentinib In STK11-mutated Advanced/Metastatic Non-small Lung Cancer (NSCLC)

BerGenBio ASA (OSE: BGBIO), a clinical-stage biopharmaceutical company developing novel, selective AXL inhibitors for severe unmet medical needs, today announces that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation for bemcentinib in combination with an anti-PD-(L)1 agent as treatment for patients with STK11 altered advanced/metastatic NSCLC patients without actionable mutations.

Fast Track designation is intended to facilitate the development and review of drugs used to treat serious conditions and to fill an unmet medical need. It will enable BerGenBio to have more frequent interactions with the FDA throughout the drug development process so that an approved product can potentially reach the market faster.

In a separate release today 9 November 2021, BerGenBio announced that in pre-clinical NSCLC mouse models harboring STK11 mutations, sensitivity to PD-1 blockade was evaluated in the absence and presence of bemcentinib. Systemic inhibition of AXL with bemcentinib resulted in the expansion of tumor-associated T cells and restored therapeutic response to anti-PD-1 check point inhibition.

Further, data from BerGenBio’s Phase II bemcentinib and pembrolizumab combination study (BGBC008) in advanced NSCLC showed that 3 of 3 evaluable patients with identified STK11/LKB1 mutations demonstrated objective clinical response / clinical benefit to the combination of AXL inhibitor bemcentinib and pembrolizumab.

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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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11-16-2021

U.S. FDA Approves Nova Mentis Orphan Drug Application

Nova Mentis Life Science Corp. (CSE: NOVA) (FSE: HN3Q) (OTCQB: NMLSF) (“NOVA” or the “Company”), a biotechnology company and global leader in first-in-class psilocybin-based therapeutics and complementary diagnostics for neuroinflammatory disorders, is pleased to announce that the U.S. FDA has approved the Company’s proprietary psilocybin drug Orphan Drug Application to  treat patients with fragile X syndrome (FXS), the most common inherited cause of autism spectrum disorder (ASD).

“NOVA has established a unique position in the field of psychedelic therapy by having its FXS program achieve orphan drug status in both the United States and European Union,”says Will Rascan, NOVA’s CEO & President. “I am pleased to announce that we are the first biotech company to have psilocybin registered for treatment of FXS in the drug regulatory logs of both the FDA and EMA.”

Medicines that have been granted an orphan designation from the U.S. Food and Drug Administration (FDA) and European Medicines Agency (EMA) receive  benefits, including but not limited to: 

  • Seven-Ten years of market exclusivity: FDA and EMA cannot approve the same drug for the same indication.
  • 25% federal tax credit for expenses incurred in conducting clinical research within the United States.
  • Waiver of Prescription Drug User Fee Act (PDUFA) fees: a value of approximately $2.9 million.
  • Ability to qualify to compete for research grants from the Office of Orphan Products Development (OOPD) to support clinical studies.
  • Eligibility to receive regulatory assistance and guidance from the FDA in the design of an overall drug development plan.

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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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11-15-2021

US FDA accepts supplemental New Drug Application and grants Priority Review for Jardiance® for adults with heart failure independent of left ventricular ejection fraction

The U.S. Food and Drug Administration (FDA) has accepted a supplemental New Drug Application (sNDA) and granted Priority Review for Jardiance® (empagliflozin) 10 mg, which is being investigated as a potential new treatment to reduce the risk of cardiovascular death plus hospitalization for heart failure in adults with heart failure independent of left ventricular ejection fraction (LVEF), Boehringer Ingelheim and Eli Lilly and Company (NYSE: LLY) announced.

“If approved, Jardiance would be the first and only therapy clinically proven to significantly improve outcomes in a heart failure population that included a majority of people with preserved ejection fraction,” said Mohamed Eid, M.D., M.P.H., M.H.A., vice president, Clinical Development & Medical Affairs, Cardio-Metabolism & Respiratory Medicine, Boehringer Ingelheim Pharmaceuticals, Inc. “Building on the recent FDA approval of Jardiance for heart failure with reduced ejection fraction, this supplemental New Drug Application acceptance is a step toward the potential to make Jardiance the sole treatment to demonstrate a statistically significant benefit for adults across the full spectrum of heart failure regardless of ejection fraction. The FDA’s Priority Review designation further reinforces the urgent need for additional treatments for heart failure.”

The sNDA is based on results from the EMPEROR-Preserved® phase III trial, in which Jardiance was associated with a 21% relative risk reduction (3.3% absolute risk reduction) for the composite primary endpoint of cardiovascular death or hospitalization for heart failure in adults with heart failure with LVEF over 40% compared with placebo. Results were independent of ejection fraction or diabetes status. Results from EMPEROR-Preserved were presented at the European Society of Cardiology Congress 2021 and published in The New England Journal of Medicine.

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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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11-14-2021

Gamida Cell Provides Update on Pre-BLA Meeting With FDA for Omidubicel

Gamida Cell Ltd. (Nasdaq: GMDA), an advanced cell therapy company committed to cures for cancer and other serious diseases, completed a Type B Pre-Biologics License Application (BLA) meeting with the U.S. Food and Drug Administration (FDA) for omidubicel, a potentially life-saving treatment for patients with blood cancers in need of stem cell transplant. The FDA requested that Gamida Cell provide revised analysis of the manufacturing data generated at Gamida Cell’s wholly-owned commercial manufacturing facility to demonstrate the comparability to the omidubicel that was produced at the clinical manufacturing sites for the Phase 3 study. The FDA did not request additional clinical data to initiate the BLA submission once analytical comparability is demonstrated. The company will continue to work collaboratively with the FDA and anticipates submitting the BLA in the first half of 2022 in lieu of the company’s previous plan to submit the BLA by the end of 2021.

“Despite the delay in timing to bring omidubicel to patients after a potential FDA approval, we are encouraged by the FDA’s reaction to our Phase 3 data as the pivotal trial of omidubicel. We have gained further clarity with the FDA on the requirements for demonstrating comparability for our commercial manufacturing facility,” said Julian Adams, Ph.D., Chief Executive Officer of Gamida Cell. “With the FDA’s feedback in hand, we believe that we are one step closer for omidubicel to be made available to patients in need.”

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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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11-11-2021

Dragonfly Therapeutics Announces DF1001 Granted Orphan Drug Designation in the US for Esophageal Cancer

Dragonfly Therapeutics, Inc., a clinical stage biotechnology company developing novel immunotherapies, today announced that its wholly-owned HER2-targeted NK cell engager therapy, DF1001, has been granted Orphan Drug Designation (ODD) in the US for treatment of esophageal cancer.

An estimated 19,260 new cases of esophageal cancer will be diagnosed this year and the disease could lead to more than 15,500 deaths in the US in 2021.1 The Food and Drug Administration (FDA) grants ODD to medicines intended for the treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 people in the US. HER2 is expressed in a large subset of esophageal cancers, even in patients without amplification of erbb2.

An ongoing Phase 1/2 clinical trial for the DF1001 TriNKET is a first-in-human study exploring the safety, tolerability and preliminary biological and clinical activity of DF1001. Dragonfly has treated 40 patients with DF1001, with no DLTs to date. Clinical trial sites are open in the U.S., France, Belgium, Denmark and The Netherlands. Additional information about the trial, including eligibility criteria, can be found at: https://clinicaltrials.gov (ClinicalTrials.gov Identifier: NCT04143711).

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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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11-10-2021

Single Blood Test Can Detect 50 Types of Cancer In Early Stages

Cancer therapy is seeing a silver lining with the launch Grail’s Galleri blood test, which claims that it has the ability to detect as many as 50 types of cancer.

The blood test has yet to gain approval from the U.S. Food and Drug Administration (FDA), which has so far issued a Breakthrough Therapy Designation, but GRAIL has already signed a deal with the National Health Service (NHS) in the U.K. to support programs that hope to detect cancer early and save more lives. 

Despite the absence of the FDA’s seal, the New York State Department of Health also issued a go-ahead to use Galleri for early cancer detection in September. The test is now available to residents by prescription, in addition to existing single cancer screening tests. 

“The approval of the New York State Department of Health marks a significant regulatory milestone for GRAIL and confirms the high standard for validation of our rigorous approach to test development, and the high quality of our clinical laboratory,” commented Dr. Josh Ofman, chief medical officer and head of external affairs at GRAIL, in a statement. 

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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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11-09-2021

Lipocine Announces FDA Grants Fast Track Designation to LPCN 1144 for Treatment of Non-Cirrhotic NASH

Lipocine Inc. (LPCN), a clinical-stage biopharmaceutical company focused on metabolic and endocrine disorders, today announced that the U.S. Food and Drug Administration (“FDA”) has granted Fast Track Designation to LPCN 1144 as a treatment for non-cirrhotic non-alcoholic steatohepatitis (“NASH”). LPCN 1144, an oral prodrug of bioidentical testosterone, was recently studied in the Liver Fat intervention with oral Testosterone (“LiFT “) Phase 2 paired biopsy clinical study in patients with confirmed NASH.  Treatments with LPCN 1144 in the LiFT clinical study resulted in robust liver fat reduction, assessed by magnetic resonance imaging, proton density fat fraction (“MRI-PDFF”) technique, and showed improvement of liver injury markers with no observed tolerability issues. Additionally, key secondary endpoint results after 36 weeks of treatment in the LiFT clinical study demonstrated that treatments with LPCN 1144 met the NASH resolution regulatory endpoint, showed positive effects in appendicular lean mass and whole-body fat mass and continued to show substantial reductions in markers of liver injury compared to placebo.

“The granting of Fast Track Designation represents an important recognition by the FDA of LPCN 1144’s potential to address a significant unmet need in the treatment of NASH,” said Dr. Mahesh Patel, Chairman, President and Chief Executive Officer of Lipocine. “We believe the Fast Track Designation will enable us to work closely with the FDA on our development program for NASH, including the design of the Phase 3 program.”

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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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