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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10-21-2021

Novian Health Gains FDA Breakthrough Designation for Novilase Breast Therapy

Novian Health Inc. has been granted Breakthrough Device designation from the U.S. Food and Drug Administration for its Novilase®Interstitial Laser System and its proposed indication for use: the focal destruction of malignant breast tumors in adult women who seek a breast conserving procedure.

The FDA’s Breakthrough Device program supports the timely development of technologies that have the potential to provide more effective treatment of life-threatening diseases. By obtaining this status, Novian will benefit from an expedited regulatory review process, which may accelerate access to its interstitial laser therapy system in the United States.

“Laser therapy can help patients avoid the trauma and risks of breast cancer surgery,” said Henry Appelbaum, president and CEO of Novian Health. “Receiving this designation is a significant milestone that moves us one step closer to offering the benefits of this game-changing technology to breast cancer patients in the U.S.”

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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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10-20-2021

AIM ImmunoTech Submits IND and Accompanying Fast Track Application for Phase 2 Trial of Ampligen in Patients with Locally Advanced or Metastatic Late-Stage Pancreatic Cancer

AIM ImmunoTech Inc. (NYSE American: AIM) today announced that it has submitted an Investigational New Drug application (IND) and an accompanying application for Fast Track status with the U.S. Food and Drug Administration (FDA) for a planned Phase 2 study of the company’s drug Ampligen as a therapy for locally advanced or metastatic late-stage pancreatic cancer.

The planned AMP-270 clinical trial of approximately 250 subjects will be a Phase 2, randomized, open-label, controlled, parallel-arm study with the primary objective of comparing the efficacy of Ampligen when added to SOC (standard of care) versus SOC alone for subjects with advanced pancreatic carcinoma recently treated with FOLFIRINOX chemotherapy regimen. Secondary objectives include comparing safety and tolerability.

Amarex Clinical Research will manage the AIM-sponsored study. The Buffett Cancer Center at the University of Nebraska Medical Center (UNMC) and Erasmus MC in The Netherlands are expected to be the primary study sites, although additional sites are anticipated.

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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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10-19-2021

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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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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10-18-2021

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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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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10-15-2021

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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10-14-2021

Takeda Racks Up Designations for Rare Lung Cancer Drug

The U.S. Food and Drug Administration (FDA) today awarded a number of approvals for Takeda Pharmaceutical’s candidate drug EXKIVITY in the treatment of adult patients with locally advanced or metastatic non-small cell lung cancer. 

Takeda was granted priority review and received a Breakthrough Therapy designation, Fast Track designation, and Orphan Drug designation, as the first and only approved oral therapy designed to target epidermal growth factor receptor (EGFR) Exon20 insertion mutations. EXKIVITY, also called mobocertinib, is a first-in-class oral tyrosine kinase inhibitor (TKI) indicated for NSCLC cases that have progressed on or after platinum-based chemotherapy. 

Patients with EGFR Exon20+ NSCLC make up around one to two percent of patients, and the disease is more common in Asian populations than in Western communities. It carries a worse prognosis compared to other EGFR mutations and current chemotherapy interventions provide little to no benefit. 

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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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10-12-2021

Alzheimer’s Drug Granted FDA Breakthrough Designation as Space Heats Up

Global biotech giant Roche has announced that the U.S. Food and Drug Administration (FDA) has awarded its candidate Alzheimer’s Disease drug gantenerumab Breakthrough Therapy Designation, bringing it closer to finally getting a full FDA approval. 

The decision is based on promising results from the ongoing Marguerite RoAD and SCarlet RoAD open-label extension trials, showing a significant reduction in brain amyloid plaque in Alzheimer’s patients. Findings from both trials have been incorporated into the ongoing global Phase III trials GRADUATE I and II. The pivotal GRADUATE trials are assessing the effect of gantenerumab in over 2,000 participants over the last two years. 

Gantenerumab is an IgG1 antibody that can remove brain amyloid plaques by binding to aggregated beta-amyloid structures. The GRADUATE trials are looking into the safety and efficacy of the drug in patients diagnosed with prodromal-to-mild Alzheimer’s Disease. The massive study covers 250 centers in over 30 countries, where patients are given a 1,020 mg dose of gantenerumab monthly with optimized titration. Both GRADUATE 1 and 2 are expected to be done by mid-2022. 

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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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10-11-2021

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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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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