Posts tagged: BTD

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.

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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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.

To Read the Complete Article at US Food & Drug Administration 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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Tetra Bio-Pharma Inc. (“Tetra” or the “Company”) (TSX: TBP) (OTCQB: TBPMF) (FRA: JAM1), a leader in cannabinoid-derived drug discovery and development today announced that the European Medicines Agency (EMA) Committee for Orphan Medicinal Products (COMP) issued a positive opinion on the Company’s application for Orphan Drug Designation (ODD) for its investigational medicine QIXLEEF™ as a potential treatment for Complex Regional Pain Syndrome (CRPS), a chronic neuropathic pain condition.

Dr. Guy Chamberland, Chief Executive Officer and Chief Regulatory Officer of Tetra Bio-Pharma said “the positive opinion issued by the COMP is excellent news as Tetra continues to execute its regulatory strategy in Europe. An ODD brings several unique advantages, from a cost reduction in drug development, to an accelerated review process and market exclusivity for 10 years. Such strategy is cost and time effective and allows the Company to easily gain market shares in a competitive free environment. If granted, this would represent QIXLEEF™’s second ODD as a potential treatment for CRPS, in addition to the ODD granted by the U.S. FDA in March 2018. We firmly believe that QIXLEEF™ will be a safe and effective medicine for pain management and an alternative to opioids.”

The positive opinion issued by the COMP will be sent to the European Commission, which is expected to grant the orphan designation within 30 days. The Committee based its opinion on Article 5 of Regulation (EC) No 141/2000 of 16 December 1999. The COMP based on majority of 30 out of 31 votes concluded that QIXLEEF™ satisfies the criteria for such designation and recommends the granting of orphan medicinal product designation.

To Read the Complete Article at US Food & Drug Administration 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, the National Institutes of Health, 10 pharmaceutical companies and five non-profit organizations have partnered to accelerate development of gene therapies for the 30 million Americans who suffer from a rare disease. While there are approximately 7,000 rare diseases, only two heritable diseases currently have FDA-approved gene therapies. The newly launched Bespoke Gene Therapy Consortium (BGTC), part of the NIH Accelerating Medicines Partnership (AMP) program and project-managed by the Foundation for the National Institutes of Health (FNIH), aims to optimize and streamline the gene therapy development process to help fill the unmet medical needs of people with rare diseases.

“By leveraging on experience with a platform technology and by standardizing processes, gene therapy product development can be accelerated to allow more timely access to promising new therapies for patients who need them most,” said Peter Marks, M.D., Ph.D., Director of the Food and Drug Administration (FDA)’s Center for Biologics Evaluation and Research. “FDA is committed to developing a regulatory paradigm that can advance gene therapies to meet the needs of patients with rare diseases.”

“Most rare diseases are caused by a defect in a single gene that could potentially be targeted with a customized or ‘bespoke’ therapy that corrects or replaces the defective gene,” said NIH Director Francis S. Collins, M.D., Ph.D. “There are now significant opportunities to improve the complex development process for gene therapies that would accelerate scientific progress and, most importantly, provide benefit to patients by increasing the number of effective gene therapies.”

To Read the Complete Article at US Food & Drug Administration 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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Novartis today announced that the US Food and Drug Administration (FDA) and the European Medicines Agency (EMA) have accepted the company’s Supplemental Biologics License Application (sBLA) and Type II Variation, respectively, for Kymriah^® (tisagenlecleucel) in adult patients with relapsed or refractory (r/r) follicular lymphoma (FL) after two prior lines of treatment. The FDA has also granted priority review to the company’s sBLA for Kymriah in adult patients with r/r FL. Kymriah was previously granted orphan medicinal product designation by the European Commission (EC) for FL. If approved in this potential third indication, Kymriah would have the opportunity to present an important treatment option for those patients with r/r FL in need of potentially definitive outcomes.

The regulatory submissions are based on positive data from the pivotal Phase II ELARA trial, which investigated the efficacy and safety of Kymriah in adult patients with r/r FL. The trial met the primary endpoint with robust responses observed in heavily pretreated patients. The safety profile was remarkable, with no patients experiencing grade 3 or higher cytokine release syndrome (CRS) related to Kymriah within the first 8 weeks following infusion¹. Data from the trial was presented earlier this year as an oral presentation during the 2021 Annual American Society of Clinical Oncology (ASCO) Virtual Scientific Meeting.

“This is an important milestone in our mission to bring Kymriah to adult patients with relapsed or refractory follicular lymphoma. Receiving orphan drug designation from the EC as well as priority review from the FDA underscores the unmet need and urgency for these patients. With Kymriah demonstrating impressive results in the ELARA trial, we are hopeful that we can offer a unique and potentially definitive treatment that minimizes the burden,” said Jeff Legos, Executive Vice President, Global Head of Oncology & Hematology Development, Novartis.

To Read the Complete Article at Novartis 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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Ambulero Inc., a biotechnology company (“Ambulero” or the “Company”) developing new cell and gene therapy treatments for patients suffering from severe vascular disease, today announced that the U.S. FDA’s Office For Orphan Products Development (OOPD) granted the company’s request for orphan drug status for its gene therapy candidate, AMB-301, to treat Buerger’s Disease (BD), also known as Thromboangiitis Obliterans (TAO).

AMB-301 is a first-in-class gene therapy vector encoding a cell adhesion molecule (E-selectin). Over a decade of research in animal models of vascular disease suggests that providing E-selectin to damaged blood vessels promotes therapeutic angiogenesis, robust tissue regeneration and improved limb function.

BD is a debilitating vascular disease that can lead to severe limb damage and amputation, often in relatively young patients. There are no effective treatments and existing therapies are largely ineffective. AMB-301 is a promising gene therapy candidate for enhancing blood vessel formation, restoring tissue integrity and eliminating the need for amputation as a treatment option.

The FDA’s orphan drug designation provides AMB-301 with seven (7) years of market exclusivity for the treatment of BD if approved. The FDA grants orphan designations to medical products showing promise to treat diseases that affect 200,000 or fewer Americans. The company is also eligible for certain tax credits and regulatory benefits with the FDA designation.

To Read the Complete Article at BioSpace 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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By combining computational and experimental approaches, University of Pittsburgh School of Medicine and Prairie View A&M University researchers identified cancer drugs that show promise for treating pulmonary hypertension, or PH, a rare and incurable lung disease.

Published today in Science Advances, the study used a new algorithm to identify candidate cancer drugs for PH. Two of these compounds improved markers of the disease in human cells and rodents. The findings support broader use of this drug-repurposing platform for other non-cancerous conditions that don’t yet have effective treatments.

“Repurposing drugs can cut down the time and cost of developing treatments for rare diseases, which historically don’t receive much investment into research and drug development,” said senior author Stephen Chan, M.D., Ph.D., professor of medicine and director of the Vascular Medicine Institute at Pitt and UPMC. “Pulmonary hypertension is an example of a rare disease where there is an unmet need for new treatments, given its devastating consequences. We developed this pipeline to rapidly predict which drugs are effective for PH and get these treatments to patients faster.”

Pulmonary hypertension is a type of high blood pressure that occurs in the vessels that transport blood from the heart to the lungs. As the disease progresses and the heart must strain harder against these high pressures, it can lead to heart failure, multi-organ dysfunction and death. PH affects people of all ages but hits young women more often than men.

To Read the Complete Article at TechnologyNetworks 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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Sio Gene Therapies Inc. (NASDAQ: SIOX), a clinical-stage company focused on developing gene therapies to radically transform the lives of patients with neurodegenerative diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track Designation to AXO-AAV-GM1, its adeno-associated viral vector (AAV)9-based gene therapy candidate for the treatment of Type I (early infantile-onset) and Type II (late infantile-onset and juvenile-onset) GM1 gangliosidosis. The Fast Track process is designed to facilitate the development and expedite the review of drugs to treat serious conditions and fill an unmet medical need.

“Receiving Fast Track Designation is a critical step in our mission to develop the first potential treatment for all pediatric forms of this rare, terminal disease. This designation joins both the Orphan Drug Designation and Rare Pediatric Disease Designation assigned to AXO-AAV-GM1 by the FDA, which we believe further demonstrates the potential impact of this work on the patient community,” said Pavan Cheruvu, M.D., Chief Executive Officer of Sio Gene Therapies. “Building on the recently presented data at ESGCT demonstrating normalization of key disease biomarkers in the high-dose cohort with no serious adverse events attributed to AXO-AAV-GM1, this designation will help us accelerate clinical development of this promising investigational therapy for children and families.”

The current Phase 1/2 study (NCT03952637) is designed to evaluate the safety, tolerability, and potential efficacy of AXO-AAV-GM1 gene therapy delivered intravenously in children with early infantile, or Type I, and late infantile and juvenile, or Type II, GM1 gangliosidosis. Stage 1 of the study is a dose-escalation study in which the low-dose cohort is evaluating 1.5×10¹³ vg/kg and the high-dose cohort is evaluating a dose of 4.5×10¹³ vg/kg. Stage 2 of the trial will then evaluate the efficacy and safety of the optimal dose identified in Stage 1.

To Read the Complete Article at Pinsent Masons 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 European Commission has begun a public consultation on potential reforms to EU general pharmaceutical law.

The review of the EU’s general pharmaceutical legislation is a core plank of the European Commission’s pharmaceutical strategy, published in November 2020. The Commission said the aim of the review is to “ensure a future-proof and crisis-resistant medicines regulatory system”.

In its review, the Commission is considering potential reform in a wide range of areas including in relation to unmet medical needs; improving access to and affordability of medicines; addressing antimicrobial resistance, ensuring security of supply of medicines, adapting the regulatory approach to account for “novel products”; and environmental challenges.

Notably, EU policymakers are considering how to stimulate innovation in areas of unmet medical needs and whether the existing incentives framework is fit for purpose. The consultation seeks views on potential changes to the current regulatory data and market exclusivities which innovators enjoy upon getting regulatory approval for new speciality medicines. The proposed options include introducing new conditions to existing incentives – such as a commitment to make the medicine available in all EU member states – granting longer periods of protection in areas of unmet medical needs, or introducing new types of incentives in these areas.

To Read the Complete Article at Pinsent Masons 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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