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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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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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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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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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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11-08-2021

Breakthrough Cervical Cancer Treatment Wins Accelerated FDA Approval

Patients with recurrent or metastatic cervical cancer whose disease has progressed on or after chemotherapy may see a ray of hope soon after the U.S. Food and Drug Administration granted accelerated approval to Genmab and Seagen’s TIVDAK drug. 

TIVDAK (tisotumab vedotin-tftv) is the first and only approved antibody-drug conjugate (ADC) for the treatment of the said disease in adults. It earned the FDA’s green light after its Phase II innovaTV 204 clinical trial delivered stellar results in terms of tumor response and durability of the response. Continued approvals are likely, but not until further confirmatory trials verify more clinical benefits. 

In the Phase II trial, TIVDAK was observed in 101 patients who had recurrent or metastatic cervical cancer and has received a maximum of two prior systemic regimens, including one prior platinum-based chemotherapy treatment. 

After an assessment by an independent review committee (IRC) using the Response Evaluation Criteria in Solid Tumors (RECIST) v1.1, TIVDAK was revealed to have an objective response rate (ORR) of 95 percent, while the median duration of response (DOR) was 8.3 months. 

Approximately 14,480 new cases of invasive cervical cancer are expected to be diagnosed this year in the U.S. alone, and 4,290 women are likely to die from the disease. It is one of the leading causes of cancer death in the world. 

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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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11-05-2021

Menarini Receives FDA Orphan Drug Designation for SEL24/MEN1703, a first in class, dual PIM/FLT3 inhibitor for the Treatment of Acute Myeloid Leukemia

The Menarini Group announced today that the U.S. Food and Drug Administration (FDA) granted orphan drug designation (ODD) to SEL24/MEN1703 for the treatment of Acute Myeloid Leukemia (AML). SEL24/MEN1703 is a first-in-class, orally available, dual PIM/FLT3 inhibitor, in-licensed by Menarini from Ryvu Therapeutics, and currently investigated in the DIAMOND-01 trial as a single agent for the treatment of patients with relapsed/refractory AML.

ODD is granted by the FDA to therapies intended for the treatment of conditions that impact fewer than 200,000 people in the US and provides companies with several incentives to support the development of therapeutics and diagnostics for rare diseases. Of note, ODD does not influence the process of regulatory approval, and drugs for rare diseases go through the same rigorous scientific review process established for any other drug.

FDA orphan drug designation represents an important milestone for SEL24/MEN1703 program,” said Elcin Barker Ergun, Chief Executive Officer of the Menarini Group. “SEL24/MEN1703 is a first-in-class, orally available, dual PIM/FLT3 inhibitor that can contribute to finding new treatment paradigms for AML where significant unmet needs exist especially as resistance develops in later lines. We look forward to advancing the clinical development of SEL24/MEN1703 in AML with a goal to ultimately provide patients with a new therapeutic option for this hard-to-treat disease.”

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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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