breakthrough therapy designation Archives

Acer Therapeutics’ EDSIVO™ (celiprolol) Granted FDA Breakthrough Therapy Designation for Vascular Ehlers-Danlos Syndrome

April 6, 2022

Acer Therapeutics Inc., a pharmaceutical company focused on the acquisition, development and commercialization of therapies for serious rare and life-threatening diseases with significant unmet medical needs, today announced the U.S. Food and Drug Administration (FDA) has granted celiprolol Breakthrough Therapy designation (BTD) in the U.S. for the treatment of patients with COL3A1-positive vascular Ehlers-Danlos syndrome (vEDS).

BTD is a process designed to expedite the development and review of drugs that are intended to treat a serious condition and preliminary clinical evidence indicates that the drug may demonstrate substantial improvement on at least one clinically significant endpoint(s) over available therapy.

“With no currently approved treatments for vEDS anywhere in the world, this designation by FDA marks an important step forward in support of our goal to provide treatment options like EDSIVO™ to rare disease patients, who are often overlooked or underserved,” said Adrian Quartel, MD, Chief Medical Officer of Acer. “We look forward to continuing our discussions with FDA, through the SPA process, to seek agreement on the protocol design of the proposed pivotal Phase 3 DiSCOVER trial that we plan to initiate by the end of Q2 2022 once agreement is reached.”

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BioPharma Global is a mission-driven corporation 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 unmet medical needs. 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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FDA Grants BI 1015550 Breakthrough Therapy Designation for Idiopathic Pulmonary Fibrosis

February 25, 2022

by administrator BPG Blog

Boehringer Ingelheim Pharmaceuticals, Inc. announced today that the U.S. Food and Drug Administration (FDA) granted Breakthrough Therapy Designation to its novel investigational therapy, BI 1015550, for the treatment of idiopathic pulmonary fibrosis (IPF). BI 1015550 is an oral, phosphodiesterase 4B (PDE4B) inhibitor with the potential to address both pulmonary fibrosis – an irreversible scarring of lung tissue that negatively impacts lung function – and inflammation associated with progressive fibrosing interstitial lung diseases (ILDs).

“The accelerated development of BI 1015550 is part of Boehringer Ingelheim’s next wave of potential innovative treatments for interstitial lung diseases aimed at preserving lung function and improving the lives of patients,” said Thomas Seck, M.D., senior vice president, Medicine and Regulatory Affairs, Boehringer Ingelheim Pharmaceuticals, Inc. “BI 1015550 represents the first molecule in the class of PDE4B inhibitors that is being studied for IPF and other progressive fibrosing ILDs. We have built on our heritage in pulmonary fibrosis and are acting on the breakthrough designation and clinical data with the goal that this potential novel medicine can reach patients as soon as possible.”

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Bavarian Nordic Announces Breakthrough Therapy Designation for its RSV Vaccine Candidate for the Prevention of Respiratory Syncytial Virus

February 14, 2022

by administrator BPG Blog

Bavarian Nordic A/S (OMX: BAVA) announced today that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation for the Company’s vaccine candidate, MVA-BN RSV, for active immunization for prevention of lower respiratory tract disease caused by respiratory syncytial virus (RSV) in adults aged 60 years or older.

A Breakthrough Therapy Designation is designed to expedite the development and regulatory review of medicines that are intended to treat a serious condition. The designation has been granted upon the assessment of preliminary clinical evidence for MVA-BN RSV, indicating that the vaccine candidate may demonstrate substantial improvement over available therapy on a clinically significant endpoint¹. The designation provides an option to work more closely with FDA for the development and review of MVA-BN RSV.

Paul Chaplin, President and CEO of Bavarian Nordic, said: “The development of an RSV vaccine is one of our main priorities that could help fulfil the significant unmet need for a preventative therapy, particularly for the elderly who may be at risk from serious complications from the disease. Our candidate has a clearly differentiated approach, as it employs five RSV-specific antigens with an aim to stimulate a broad antibody and cellular immune response against RSV. Based on the encouraging results demonstrated in a human challenge trial last year, we recently announced our plans to initiate a Phase 3 study this year and look forward to working closely with the FDA on our path towards licensure of the vaccine.”

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FDA Grants Breakthrough Therapy Designation for Dizal Pharmaceutical’s DZD9008 in Patients with Non-Small Cell Lung Cancer Harboring EGFR Exon20 Insertion

January 31, 2022

by administrator BPG Blog

Dizal pharma Co., Ltd. (SHEX:688192) (“Dizal”), today announced that the U.S. Food and Drug Administration (“FDA”) has granted Breakthrough Therapy Designation to DZD9008 (Sunvozertinib) for the treatment of patients with locally advanced or metastatic non-small cell lung cancer (NSCLC) with epidermal growth factor receptor (EGFR) exon20 insertion mutations whose disease has progressed on or after platinum-based chemotherapy.

“Lung cancer patients with exon20 insertion mutations need better treatment. Sunvozertinib was specifically designed with high selectivity for inhibiting mutated EGFR, which causes cancers. Available clinical evidence shows that Sunvozertinib has the potential to provide the patients with a new and much improved targeted therapy,” said Dr. Xiaolin Zhang, Chief Executive Officer at Dizal. “We are very pleased with FDA’s decision. Now Sunvozertinib has received Breakthrough Therapy Designation from both US FDA and China CDE, which validates Sunvozertinib’s differentiated profile. At Dizal, we are committed to discover and develop innovative medicines for the benefit of cancer patients globally.”

Breakthrough Therapy designation is a process designed by US FDA, China CDE, and other regulatory agencies to expedite the development and review of drugs that are intended to treat a serious condition, and preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over available therapy on a clinically significant endpoint(s)

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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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FDA Grants Breakthrough Therapy Designation for Cullinan Oncology’s CLN-081 in Patients with Non-Small Cell Lung Cancer

January 7, 2022

by administrator BPG Blog

Cullinan Oncology (Nasdaq: CGEM) (“Cullinan”), a biopharmaceutical company focused on developing a diversified pipeline of targeted therapies for cancer patients, today announced that the U.S. Food and Drug Administration (“FDA”) has granted Breakthrough Therapy Designation for CLN-081 for the treatment of patients with locally advanced or metastatic non-small cell lung cancer (“NSCLC”) harboring epidermal growth factor (“EGFR”) exon 20 insertion mutations who have previously received platinum-based systemic chemotherapy.

“We are extremely pleased that Cullinan has received breakthrough therapy designation from the FDA for CLN-081, a distinction that underscores the urgent need to bring improved targeted treatments to this patient population and further supports the differentiated clinical profile of CLN-081,” said Nadim Ahmed, Chief Executive Officer of Cullinan Oncology. “The updated data from our ongoing Phase 1/2a study in a larger number of patients have demonstrated a high response rate with durable responses and encouraging progression free survival in heavily pre-treated patients. We are also encouraged by the favorable safety profile observed thus far, and we look forward to ongoing, productive regulatory discussions with the FDA, which are further enabled with this designation.”

Breakthrough Therapy Designation is a process designed to expedite the development and review of drugs that are intended to treat a serious condition and preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over available therapy on a clinically significant endpoint(s).

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NRx Pharmaceuticals Files Breakthrough Therapy Designation Request for ZYESAMI® in Patients at Immediate Risk of Death from COVID-19 Despite Treatment

December 29, 2021

by administrator BPG Blog

NRx Pharmaceuticals (Nasdaq: NRXP) announced today that it has filed a new Breakthrough Therapy Designation (BTD) request with the US Food and Drug Administration (FDA) focused on patients with Critical COVID-19 and respiratory failure who are at immediate risk of death despite treatment with remdesivir and other approved therapies.

NRx filed the request after the FDA’s request for clinical data on ZYESAMI® vs. Remdesivir in these high risk patients. Prof. David Schoenfeld, one of the world’s most widely published statisticians with unique expertise in life-threatening diseases of the lung conducted the new analysis.

ZYESAMI® (aviptadil) has demonstrated a statistically significant two-fold increased odds of survival compared to placebo across all patients and hospitals studied in a randomized trial of 196 patients. However, in 70% of patients who were already treated with Remdesivir and continued to progress despite all approved therapies, ZYESAMI has demonstrated a highly significant four-fold increased odds of survival compared to placebo at 60 days (P=.006). Moreover, those treated with ZYESAMI after Remdesivir has failed demonstrate a 3-fold increased odds of being both alive and free of respiratory failure at both 28 and 60 days, compared to placebo (P=.03).

The FDA recently declined Emergency Use Authorization (EUA) and Breakthrough Therapy Designation (BTD) for ZYESAMI and invited a new request based on new clinical evidence that aviptadil may demonstrate a significant improvement in treatment over existing therapies. Based on the FDA’s input, NRx has narrowed its BTD request to treatment of COVID-19 respiratory failure in patients who progress despite treatment with remdesivir and other approved therapies.

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Patritumab Deruxtecan Granted FDA Breakthrough Therapy Designation for Metastatic EGFR-Mutated Non-Small Cell Lung Cancer

December 26, 2021

by administrator BPG Blog

Daiichi Sankyo Company, Ltd. (hereafter, Daiichi Sankyo) announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation (BTD) to patritumab deruxtecan (HER3-DXd), a potential first-in-class HER3 directed antibody drug conjugate (ADC), for the treatment of patients with metastatic or locally advanced EGFR-mutated non-small cell lung cancer (NSCLC) with disease progression on or after treatment with a third-generation tyrosine kinase inhibitor (TKI) and platinum-based therapies.

Lung cancer is the second most common cancer and the leading cause of cancer-related mortality worldwide, with 80% to 85% classified as NSCLC.^1,2,3 While the efficacy of targeted therapy with EGFR TKIs is well-established in the treatment of advanced EGFR-mutated NSCLC, which comprises approximately 30% of patients, the development of a broad range of resistance mechanisms commonly leads to disease progression.^4,5,6After failure of an EGFR TKI, platinum-based chemotherapy has limited efficacy with progression-free survival (PFS) of approximately 4.4 to 6.4 months.⁷ Subsequent salvage therapies after EGFR TKI and platinum-based chemotherapy have PFS of 2.8 to 3.2 months.⁸

The U.S. FDA’s BTD is designed to accelerate the development and regulatory review of potential new medicines that are intended to treat a serious condition and address a significant unmet medical need. The new medicine needs to have shown encouraging preliminary clinical results that demonstrate substantial improvement on a clinically significant endpoint over available medicines.

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Cytokinetics Receives Breakthrough Therapy Designation from FDA for Aficamten for the Treatment of Symptomatic Obstructive Hypertrophic Cardiomyopathy

December 20, 2021

by administrator BPG Blog

Cytokinetics, Inc., today announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation for aficamten for the treatment of symptomatic obstructive hypertrophic cardiomyopathy (oHCM). Aficamten is a next-generation cardiac myosin inhibitor in development for the potential treatment of HCM.

Breakthrough Therapy Designation is granted by FDA to expedite the development and review of drugs that are intended to treat a serious condition when preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over available therapies on a clinically significant endpoint. A drug that receives Breakthrough Therapy Designation is eligible for all Fast Track designation features, intensive guidance on an efficient drug development program, and organizational commitment involving senior managers.

“We are pleased that the FDA has granted Breakthrough Therapy Designation for aficamten for the treatment of symptomatic oHCM, a disease associated with myriad symptoms and functional limitations,” said Fady I. Malik, M.D., Ph.D., Cytokinetics’ Executive Vice President of Research & Development. “The results of REDWOOD-HCM met our high expectations supporting the potential role of aficamten as an innovative therapy to address the hypercontractility that underlies oHCM. With start-up activities underway in SEQUOIA-HCM, our Phase 3 clinical trial of aficamten, we look forward to its further development and engaging with FDA as may hopefully benefit patients whose lives are severely impacted by this disease.”

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Longeveron Granted Orphan Drug Designation by FDA for Lomecel-B to Treat Infants with Hypoplastic Left Heart Syndrome (HLHS)

December 7, 2021

by administrator Drug Discovery News Rare Disease Highlight Rare Disease News Regulatory News

Longeveron Inc. (NASDAQ: LGVN) (“Longeveron” or “Company”), a clinical stage biotechnology company developing cellular therapies for chronic aging-related and certain life-threatening conditions, announced today that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) for Lomecel-B for the treatment of Hypoplastic Left Heart Syndrome (HLHS), a rare and life-threatening congenital heart defect in infants.

ODD is intended to assist and encourage companies to develop safe and effective therapies for the treatment of rare diseases or conditions. ODD positions Longeveron to be able to potentially leverage a range of financial and regulatory benefits, including government grants for conducting clinical trials, waiver of FDA user fees for the potential submission of a marketing application, and certain tax credits. Receiving ODD may also result in the product receiving seven years market exclusivity upon approval for use in the rare disease or condition for which the product was designated if all of the statutory and regulatory requirements are met.

“Adding to the Rare Pediatric Disease (RPD) designation already granted to Lomecel-B for treatment of HLHS, the FDA’s decision to grant ODD to Lomecel-B for this indication indicates the ongoing and unmet need for new therapies to treat infants with HLHS,” said Geoff Green, Chief Executive Officer at Longeveron. “Building on results from our completed Phase 1 safety-focused trial, we believe Lomecel-B has potential to improve outcomes for these severely impacted infants by way of repairing cardiac tissue and improving ventricular function. The combination of both RPD and ODD allows us to potentially move more efficiently through clinical development and regulatory review, and Lomecel-B may be eligible for a period of marketing exclusivity upon approval for this indication.”

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Soligenix Granted Pediatric Investigational Plan Waiver for HyBryte™ in CTCL in the United Kingdom

December 6, 2021

by administrator Drug Discovery News Rare Disease Highlight Rare Disease News Regulatory News

Soligenix (Nasdaq: SNGX) (Soligenix or the Company), a late-stage biopharmaceutical company focused on developing and commercializing products to treat rare diseases where there is an unmet medical need, announced today that it has been granted a Pediatric Investigation Plan (PIP) product-specific waiver from the Medicines and Healthcare products Regulatory Agency (MHRA) for HyBryte™ (SGX301 or synthetic hypericin), which has successfully concluded a Phase 3 pivotal clinical study for the treatment of early stage cutaneous T-cell lymphoma (CTCL).

The waiver was provided for all subsets of the pediatric population from birth to less than 18 years of age on the grounds that clinical studies in this rare population are not feasible. Earlier this year the European Medicines Agency (EMA) also granted a waiver to the Pediatric Investigational Plan requirements for the European Union (EU). With the withdrawal of the United Kingdom (UK) from the EU effective January 1, 2021, the MHRA became the UK’s standalone medicines and medical devices regulator.

“This achievement is an important regulatory milestone as we move forward with marketing applications worldwide,” stated Christopher J. Schaber, PhD, President and Chief Executive Officer of Soligenix. “The PIP waiver allows us to work towards advancing a marketing authorization application (MAA) in the UK in a more cost-effective manner since we will not need to expend resources to conduct a pediatric clinical study.”

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