Posts tagged: gene therapy

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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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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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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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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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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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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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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Approximately 30 million people in the United States—1 in 10 Americans—have been diagnosed with a rare disease, approximately two-thirds of whom are children.¹ Rare disease drugs and cell or gene therapies to treat these conditions offer new hope for people living with a rare condition and are among the fastest growing areas of the drug pipeline.

Although these treatments have the potential to save lives, they also represent the most dynamic and costly area in medicine. Complex in their administration, handling and care, these high-touch therapies require customized management and services beyond what traditional pharmacies can provide.

Serving Rare Disease Patients

Rare disease specialty pharmacies offer customizable pharmacy and patient services to make it easier for people living with rare, complex, and often isolating conditions to access the therapy, care, and resources they need. Patients prescribed advanced therapies may face unique difficulties, such as upwards of a 5-year diagnostic journey, challenges with social determinants, and access to physicians and specialists familiar with their disease.

Further, advanced therapies are often complex to manage and have a very high cost. Because of this, patients with rare diseases require higher levels of service and support.

Working with a rare disease specialty pharmacy enables a single point of contact to serve as the patient’s and caregiver’s liaison to services, education, and support. This same contact forms a relationship with the patient’s provider or prescribing office, and ensures that these providers, patients, and families have the additional support they need by coordinating a team of clinical access and reimbursement specialists, pharmacists, and ancillary health providers.

Through tailored programs for each therapy and disease, along with concierge patient-centric service, rare disease specialty pharmacies streamline the treatment process and support so patients can focus on achieving their best health care outcomes.

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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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Orphazyme A/S (ORPHA.CO; ORPH), a late-stage biopharmaceutical company, today provides an update on the regulatory status in the United States of its investigational product arimoclomol for Niemann-Pick disease type C (NPC) following a recently held Type A meeting with the U.S. Food and Drug Administration (FDA).

The Company had a collaborative dialogue with the FDA during the Type A meeting, which was held in order to discuss the key topics in the Complete Response Letter (CRL) issued by the FDA in June 2021. The CRL was issued based on the need for additional confirmatory evidence as well as additional qualitative and quantitative evidence to further substantiate the validity of the 5-domain NPC Clinical Severity Scale (NPCCSS), in particular the swallow domain, in the context of a lack of significant findings when using the FDA’s preferred and recommended statistical approach.

The Type A meeting resulted in the following take-aways:

• The FDA recommended that the Company submit additional data, information, and analyses to address certain topics in the CRL and engage in further interactions with the FDA to identify a pathway to resubmission.
• The FDA concurred with the Company’s proposal to remove the cognition domain from the NPCCSS endpoint, with the result that the primary endpoint is permitted to be recalculated using the 4-domain NPCCSS, subject to the submission of additional requested information which the Company intends to provide. To bolster the confirmatory evidence already submitted, the FDA affirmed that it would require additional in vivo or pharmacodynamic (PD)/pharmacokinetic (PK) data; the Company is considering the optimal path forward to address the FDA’s requests.

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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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