12-23-2021

Syndax Announces Orphan Drug Designation Granted to SNDX-5613 by European Commission for the Treatment of Acute Myeloid Leukemia
by BPG Blog

Syndax Pharmaceuticals Inc.   (“Syndax,” the “Company” or “we”) (Nasdaq: SNDX), a clinical stage biopharmaceutical company developing an innovative pipeline of cancer therapies, today announced that the European Commission has granted Orphan Drug Designation to SNDX-5613, the Company’s highly selective oral menin inhibitor, for the treatment of acute myeloid leukemia (AML).

“Supported by a growing body of clinical data, we firmly believe that SNDX-5613 is ideally positioned to serve as a best-in-class, meaningful intervention for patients with NPM1 and MLLr acute leukemias,” said Briggs W. Morrison, M.D., Chief Executive Officer of Syndax. “Receipt of Orphan Drug Designation from the European Commission further validates the important role that SNDX-5613 could play in the treatment of this highly underserved patient population, and we are fully committed to ensuring that it is able to reach as many of these patients as possible.”

The European Commission grants Orphan Drug Designation for medicinal products intended to treat life-threatening or chronically debilitating conditions that affect fewer than five in 10,000 people in the European Union (EU) and when no satisfactory method of diagnosis, prevention, or treatment of the condition can be authorized. The designation provides certain benefits and incentives in the EU, including protocol assistance, fee reductions, and ten years of market exclusivity once the medicine is on the market.

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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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12-22-2021

Vivoryon Therapeutics Receives FDA Fast Track Designation for Varoglutamstat in Early Alzheimer’s Disease
by BPG Blog

Vivoryon Therapeutics N.V. (Euronext Amsterdam: VVY; NL00150002Q7) (Vivoryon), a clinical stage company focused on discovery and development of small molecule medicines to modulate the activity and stability of pathologically altered proteins, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation for varoglutamstat (PQ912), an investigational oral small molecule medicine for the potential treatment of early Alzheimer’s disease (AD). Varoglutamstat is Vivoryon’s lead product candidate and is designed to block formation of N3pE amyloid, a particularly neurotoxic variant of the Abeta peptide, by inhibiting glutaminyl cyclase (QPCT) and its isoenzyme (QPCTL). Varoglutamstat is currently being investigated in two Phase 2 clinical trials in patients living with early and mild AD: the European Phase 2b VIVIAD study and the recently initiated Phase 2a/b VIVA-MIND study in the U.S.

“Having been granted Fast Track designation for varoglutamstat in early AD is extremely encouraging and we value the opportunity to interact closely with the FDA as we progress varoglutamstat through clinical development in the U.S.,” said Dr. Ulrich Dauer, CEO of Vivoryon. “We fully recognize the dire need for safe and widely available therapies to treat this devastating disease and remain dedicated to contributing to the global effort of improving the lives of the millions of patients, families and caregivers affected.”

Fast Track is a process designed to facilitate the development, and expedite the review of drugs with the potential to treat serious conditions and fill an unmet medical need, aiming to bring important new drugs to the patient earlier. With Fast Track designation, the development of varoglutamstat can benefit from more frequent engagement with the FDA to discuss varoglutamstat’s development plan and ensure collection of the appropriate data needed to successfully advance varoglutamstat through clinical development. A drug that receives Fast Track designation is also potentially eligible for Accelerated Approval and Priority Review, if relevant criteria are met.

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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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12-21-2021

Mitotech Granted Orphan Drug Designation by FDA for Visomitin in LHON
by BPG Blog

Mitotech S.A, a Luxembourg-based clinical-stage biotechnology company in ophthalmology, announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation (ODD) for the company’s topical cardiolipin peroxidation inhibitor, Visomitin, for the treatment of Leber’s Hereditary Optic Neuropathy (LHON), a rare inherited condition that can lead to blindness. Mitotech is planning to start a Phase 2 with Visomitin in LHON in 2022 in collaboration with the LHON clinic at the Doheny Eye Institute at the University of California Los Angeles (UCLA).

Visomitin demonstrated consistent improvement in visual acuity over several years in LHON patients involved in a three-year open-label Phase 2a study conducted outside the United States. Improvements were seen in patients with a range of underlying mutations, including those with variants such as G11778A where the chances of improvement are low. Mitotech’s planned Phase 2 study aims to develop Visomitin as a convenient and potentially high impact treatment for LHON.

“The US Orphan Drug Designation is highly encouraging for our efforts to bring Visomitin to young people whose lives have been radically affected by LHON,” said Natalia Perekhvatova, Chief Executive Officer of Mitotech S.A. “Visomitin has an outstanding safety profile and our preliminary human studies have demonstrated effectiveness in LHON. We are looking forward to progressing the drug in LHON as part of our wider ophthalmology pipeline.”

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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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12-20-2021

Cytokinetics Receives Breakthrough Therapy Designation from FDA for Aficamten for the Treatment of Symptomatic Obstructive Hypertrophic Cardiomyopathy
by 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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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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12-17-2021

FDA grants Calliditas Therapeutics Accelerated Approval of TARPEYO™ to Reduce Proteinuria in IgA Nephropathy
by BPG Blog

Calliditas Therapeutics AB (Nasdaq: CALT) (Nasdaq Stockholm: CALTX) (“Calliditas”) today announced that the US Food and Drug Administration (FDA) has approved TARPEYO (budesonide) delayed release capsules to reduce proteinuria in adults with primary immunoglobulin A nephropathy (IgAN) at risk of rapid disease progression, generally a urine protein-to-creatinine ratio (UPCR) ≥1.5g/g. This indication is approved under accelerated approval. It has not been established whether TARPEYO slows kidney function decline in patients with IgAN. Continued approval may be contingent upon verification and description of clinical benefit in a confirmatory clinical trial.1

This approval marks the successful transition for Calliditas to a commercial-stage biopharmaceutical company.

“We are very excited to bring the first and only FDA-approved treatment to reduce proteinuria in IgAN to market,” said Renée Aguiar-Lucander, Chief Executive Officer of Calliditas. “TARPEYO represents an FDA approved product to help these patients who are at risk of rapid disease progression.”

TARPEYO is approved under accelerated approval based on achieving its primary endpoint of reduction in proteinuria in Part A of the NeflgArd pivotal Phase 3 study, an ongoing, randomized, double-blind, placebo-controlled, multicenter study conducted to evaluate the efficacy and safety of TARPEYO 16 mg once daily vs placebo in adult patients with primary IgAN.1 The effect of TARPEYO was assessed in patients with biopsy-proven IgAN, eGFR ≥35 mL/min/1.73 m2, and proteinuria (defined as either ≥1 g/day or UPCR ≥0.8 g/g) who were on a stable dose of maximally-tolerated RAS inhibitor 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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12-15-2021

Nuvation Bio Receives FDA Fast Track Designation for NUV-422 for the Treatment of High-Grade Gliomas, Including Glioblastoma Multiforme
by BPG Blog

Nuvation Bio (NYSE: NUVB), a biopharmaceutical company tackling some of the greatest unmet needs in oncology by developing differentiated and novel therapeutic candidates, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to NUV-422, a cyclin-dependent kinase (CDK) 2/4/6 inhibitor, for the treatment of patients with high-grade gliomas, including glioblastoma multiforme. NUV-422 received Orphan Drug Designation for the treatment of patients with malignant gliomas from the FDA in the first quarter of 2021.

“We are pleased that NUV-422 received FDA Fast Track designation because it highlights the serious unmet need of patients with brain cancer and the potential of our lead drug candidate NUV-422 to serve as an innovative new treatment option for high-grade gliomas, including glioblastoma multiforme,” said David Hung, M.D., founder, president, and chief executive officer of Nuvation Bio. “Enrollment is ongoing in our expanded Phase 1/2 monotherapy study of NUV-422 for the treatment of adults with recurrent or refractory high-grade gliomas and solid tumors. We look forward to continuing to work closely with the FDA to expedite the development of NUV-422 with data from the Phase 1 portion of the study, which is on track for 2022.”

NUV-422 is the Company’s lead investigational CDK2/4/6 inhibitor program that works to overcome the limitations of CDK4/6 inhibitors. CDK4/6 inhibitors are known clinical entities with proven efficacy, but some cancer cells can evade these treatments by increasing signaling through CDK2. Inhibition of CDK2 in addition to CDK4/6 cuts off the tumor’s natural escape route. NUV-422 is a potent inhibitor of CDK 2, 4, and 6. Preclinical studies have shown that NUV-422 has favorable blood-brain barrier penetration. NUV-422 is also designed to limit CDK1 inhibition, a potential cause of toxicity in other second-generation inhibitors. In addition to high grade gliomas, NUV-422 is currently being studied in HR+ HER2- advanced breast cancer (with and without brain metastases) and metastatic castration resistant prostate cancer (mCRPC). Fast Track designation is designed to facilitate the development and expedite the review of drugs to treat serious conditions and fill an unmet medical need with the goal of getting important new drugs to patients earlier. A drug that receives Fast Track designation is eligible for more frequent meetings and communications with the FDA, accelerated approval and priority review if certain criteria are met, and more.

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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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12-14-2021

European Medicines Agency Grants Jaguar Health Orphan Drug Designation for Crofelemer to Treat Short Bowel Syndrome
by BPG Blog

Jaguar Health (NASDAQ:JAGX) and its majority owned Italian subsidiary, Napo EU S.p.A. (soon to be renamed Napo Therapeutics), today announced that the European Commission has adopted the decision to grant Orphan Drug Designation (ODD) for crofelemer for the indication of short bowel syndrome (SBS) in the European Union following review of the ODD application Napo EU submitted to the European Medicines Agency (EMA) this past September.

SBS affects approximately 10,000 to 20,000 people in the U.S.1, according to the Crohn’s & Colitis Foundation, and it is estimated that the population of SBS patients in Europe is approximately the same size.2 Despite limited treatment options, the global SBS market exceeded $568 million in 2019 and is expected to reach $4.6 billion by 2027, according to a report by Vision Research Reports.

“Following this very welcome decision from the EMA, Napo EU is initiating efforts to plan and commence a pivotal clinical trial of crofelemer in both adult and pediatric SBS patients in support of our key focus on pursuing the EMA’s accelerated conditional marketing authorization pathway in Europe for this debilitating rare disease,” said Massimo Mineo, General Manager of Napo EU.

“SBS is a devastating disease. The mortality rate of SBS patients on home parenteral nutrition is about 30% after 5 years3, and it is estimated that approximately 70% of SBS patients in Europe are pediatric,” stated Lisa Conte, Jaguar’s president and CEO and Napo EU board member. “The accelerated approval regulatory pathway in Europe is key to our strategy underlying the exclusive license Jaguar Health has provided to Napo EU for crofelemer and to our efforts to make crofelemer’s novel mechanism of action available to patients suffering from rare diseases like SBS in the most rapid manner. Additionally, Jaguar Health has the right to utilize any clinical or regulatory data generated by Napo EU for SBS on a global basis, which provides us with another potential and important ‘shot on goal’ for crofelemer.”

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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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