10-22-2021

EU considers reform of general pharma law

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.

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10-19-2021

FDA Grants Fast Track Designation to AOC 1001 for the Treatment of Myotonic Dystrophy Type 1

Avidity Biosciences, Inc. (Nasdaq: RNA), a biopharmaceutical company committed to delivering a new class of RNA therapeutics called Antibody Oligonucleotide Conjugates (AOCs™), today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track Designation to its lead program, AOC 1001, for the treatment of myotonic dystrophy type 1 (DM1).

Fast Track Designation enables more frequent interactions with the FDA to expedite the development and review process for drugs intended to treat serious or life-threatening conditions and that demonstrate the potential to address unmet medical needs.

“DM1 is an underrecognized, progressive and often fatal disease with no therapeutic options. Fast Track designation for AOC 1001 underscores this unmet need and allows us to expeditiously work with FDA to potentially deliver this first-in-class therapy to people living with DM1 as quickly as possible,” said Sarah Boyce, president and CEO of Avidity.

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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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10-18-2021

FDA grants Jardiance® Breakthrough Therapy designation for heart failure with preserved ejection fraction

The U.S. Food and Drug Administration (FDA) granted Breakthrough Therapy designation for Jardiance® (empagliflozin) as an investigational treatment for adults with heart failure with preserved ejection fraction (HFpEF), Boehringer Ingelheim and Eli Lilly and Company (NYSE: LLY) announced today.

The decision is based on results from the landmark EMPEROR-Preserved phase III trial, in which Jardiance demonstrated a 21% relative risk reduction for the composite primary endpoint of cardiovascular death or hospitalization for heart failure in adults with heart failure with preserved ejection fraction compared with placebo. The benefit was independent of ejection fraction or diabetes status. Results from EMPEROR-Preserved were presented at the European Society of Cardiology Congress 2021 on August 27 and published in The New England Journal of Medicine.

HFpEF accounts for approximately half of the more than 6 million heart failure cases in the U.S. No currently approved treatments have been clinically proven to significantly improve outcomes specifically for people with HFpEF.

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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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10-15-2021

Egetis Therapeutics receives FDA Fast Track Designation for Emcitate® for MCT8 deficiency

The FDA’s Fast Track process is designed to facilitate the development and expedite the review of new drugs intended to treat serious or life-threatening conditions to address an unmet medical need with the purpose of getting important new drugs to the patient earlier. Fast Track designation provides a company early and more frequent communication with the FDA to improve the efficiency of the investigational drug’s development and provides eligibility for priority review if certain criteria are met.

“This Fast Track Designation is an acknowledgement from the FDA of the importance of Emcitate to address the significant unmet medical need in MCT8 deficiency and ultimately help these patients that suffer from this rare and devastating disease. These patients are currently left without any treatment options. With a Fast Track designation come opportunities to expedite both the NDA submission and FDA’s review which can enable Emcitate’s regulatory approval sooner” said Nicklas Westerholm, CEO of Egetis Therapeutics.

A first clinical trial in patients suffering from MCT8 deficiency of all ages has been concluded with significant and clinically relevant results. A pivotal clinical trial looking at early intervention in very young MCT8 deficient children is under way. Recruitment is expected to be completed in Q4 2021. Emcitate has been granted Orphan Drug Designation in the US and EU. Emcitate was also granted US Rare Pediatric Disease Designation in November 2020, eligible to apply for a Priority Review Voucher.

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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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10-13-2021

Enzyvant Receives FDA Approval for RETHYMIC® (allogeneic processed thymus tissue-agdc), a One-Time Regenerative Tissue-Based Therapy for Pediatric Congenital Athymia

Enzyvant today announced the U.S. Food and Drug Administration (FDA) approval of RETHYMIC® (allogeneic processed thymus tissue-agdc), a one-time regenerative tissue-based therapy for immune reconstitution in pediatric patients with congenital athymia.

“For too long, families have faced a reality that the brutal journey for pediatric congenital athymia patients receiving supportive care only would end tragically. The FDA approval of RETHYMIC will help patients access this desperately needed therapy beyond clinical study,” said Rachelle Jacques, CEO of Enzyvant. “We are deeply grateful to the 105 patients who participated in clinical trials, their families, and all of the stakeholders who contributed to this pioneering regenerative medicine research program.”

Pediatric congenital athymia is ultra-rare with an estimated incidence of about 17 to 24 live births each year in the United States. Children who have this condition are born without a thymus and therefore have profound immunodeficiency, life-threatening immune dysregulation, and high susceptibility to potentially fatal infections. With only supportive care, children with congenital athymia typically die by age two or three.

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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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10-11-2021

Tezepelumab granted Orphan Drug Designation in the US for eosinophilic esophagitis

Tezepelumab has been granted Orphan Drug Designation (ODD) in the US by the Food and Drug Administration (FDA) for the treatment of eosinophilic esophagitis (EoE).

Tezepelumab is being developed by AstraZeneca in collaboration with Amgen and is under Priority Review for patients with asthma in the US. The FDA grants ODD status to medicines and potential new medicines intended for the treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 people in the US.

EoE is a rare, chronic, inflammatory disease that occurs when eosinophils, a type of white blood cell, accumulate in the esophagus. In addition to eosinophils, other cells including mast cells, T-cells and fibroblasts drive injury, inflammation and detrimental tissue remodelling. If the disease is not effectively treated it can make eating difficult or uncomfortable, potentially leading to chronic pain, difficulty swallowing, poor growth, malnutrition and weight loss.

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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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10-08-2021

NuCana Receives Fast Track Designation from the U.S. Food and Drug Administration for Acelarin® (NUC-1031) for the Treatment of Biliary Tract Cancer

NuCana plc (NASDAQ: NCNA) announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to Acelarin (NUC-1031), currently being evaluated in a Phase III study (NuTide:121) for the first-line treatment of patients with advanced biliary tract cancer. Fast Track is a process designed to facilitate the development and expedite the review of drugs to treat serious conditions and address an unmet medical need.

“We are very pleased that the FDA recognizes the potential of Acelarin to address the significant unmet need of patients with biliary tract cancer,” said Hugh S. Griffith, NuCana’s Founder and Chief Executive Officer. “We recently announced enrollment of 418 evaluable patients in our Phase III study, which is expected to enable the first interim analysis in the first half of 2022. This has the potential to allow for an accelerated approval of a new drug application (NDA) for Acelarin in the United States. With both Fast Track and Orphan Drug designations in place, we look forward to working closely with the FDA in our efforts to gain approval for Acelarin as the first approved front-line treatment option for patients with biliary tract cancer.”

About Fast Track Designation

Fast track is a process designed to facilitate the development, and expedite the review of drugs to treat serious conditions and fill an unmet medical need. The purpose is to get important new drugs to the patient earlier. Filling an unmet medical need is defined as providing a therapy where none exists or providing a therapy which may be potentially better than available therapy. Once a drug receives Fast Track designation, early and frequent communication between the FDA and a drug company is encouraged throughout the entire drug development and review process. The frequency of communication assures that questions and issues are resolved quickly, often leading to earlier drug approval and access by patients.

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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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10-07-2021

FDA Grants Orphan Drug Designation (ODD) to AlloVir’s Posoleucel, an Allogeneic, Off-the-Shelf, Multi-Virus Specific T-Cell Therapy, for the Treatment of Virus-Associated Hemorrhagic Cystitis

AlloVir, Inc. (Nasdaq: ALVR), a late clinical-stage cell therapy company, today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to posoleucel (Viralym-M, ALVR105) for the treatment of virus-associated hemorrhagic cystitis (HC). Posoleucel is an investigational, allogeneic, off-the-shelf, multi-virus specific T cell therapy under development for the treatment and prevention of serious diseases caused by six devastating viral pathogens: BK virus (BKV) and the related polyomavirus JC virus (JCV), cytomegalovirus (CMV), human herpes virus-6 (HHV-6), Epstein Barr virus (EBV) and adenovirus (AdV).

An inflammatory disease of the bladder, virus-associated HC is a serious complication of hematopoietic stem cell transplantation (HSCT) that can significantly prolong hospitalization and increase mortality and for which there are currently no approved or effective antiviral treatment options. The company’s Phase 3 study of posoleucel for the treatment of virus-associated HC is ongoing and enrolling adult and pediatric patients following allogeneic HSCT (allo-HSCT).

“This Orphan Drug Designation acknowledges the urgent need for new treatment options for patients who have undergone hematopoietic stem cell transplantation and are at risk for developing viral infections and hemorrhagic cystitis,” said Ercem Atillasoy, M.D., Chief Regulatory and Safety Officer, AlloVir. “We look forward to working with the FDA and regulators around the globe as we advance this therapy for 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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10-06-2021

Zai Lab Partner Turning Point Therapeutics Granted Breakthrough Therapy Designation for Repotrectinib Treatment in Patients With NTRK-Positive, TKI-Pretreated Advanced Solid Tumors

 Zai Lab Limited (NASDAQ: ZLAB; HKEX: 9688) partner Turning Point Therapeutics, Inc. (NASDAQ: TPTX), a precision oncology company developing next-generation therapies that target genetic drivers of cancer, today announced the U.S. Food and Drug Administration (FDA) granted a seventh regulatory designation to lead drug candidate, repotrectinib.

Breakthrough Therapy designation was granted for the treatment of patients with advanced solid tumors that have an NTRK gene fusion who have progressed following treatment with one or two prior TRK tyrosine kinase inhibitors, with or without prior chemotherapy, and have no satisfactory alternative treatments. The company is planning to discuss next steps towards potential registration of repotrectinib in this patient population at a Type B meeting with the FDA anticipated in the first half of 2022.

Breakthrough Therapy designation is granted by the FDA to expedite the development and regulatory review of an investigational medicine that is intended to treat a serious or life-threatening condition. The criteria for Breakthrough Therapy designation require preliminary clinical evidence that demonstrates the drug may have substantial improvement on at least one clinically significant endpoint over available therapy.

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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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10-06-2021

ImmixBio Announces FDA Orphan Drug Designation for IMX-110 for the Treatment of Soft Tissue Sarcoma

 Immix Biopharma, Inc  (“ImmixBio”), a biotechnology company pioneering Tissue Specific Therapeutics (TSTx)TM for oncology and inflammation, today announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation (ODD) to IMX-110 for the treatment of soft tissue sarcoma.

The FDA’s Office of Orphan Products Development grants orphan designation status to drugs and biologics that are intended for the safe and effective treatment, diagnosis or prevention of rare diseases, or conditions that affect fewer than 200,000 people in the U.S. Orphan Drug Designation provides certain benefits, including financial incentives, to support clinical development and the potential for up to 7 years of market exclusivity in the U.S. upon regulatory approval.

“We are pleased to receive FDA’s orphan drug designation in soft tissue sarcoma for IMX-110, the first clinical-stage product of our SMARxT Tissue-Specific Platform,” said Ilya Rachman MD PhD, ImmixBio Founder & CEO. “We are thrilled to potentially contribute to expanding therapeutic options for oncology patients, beginning with soft tissue sarcoma.”

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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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