10282020

Taysha Gene Therapies Receives Rare Pediatric Disease Designation and Orphan Drug Designation for TSHA-104 to Treat SURF1-Associated Leigh Syndrome

  • Taysha anticipated to submit Investigational New Drug Application for TSHA-104 to FDA in 2021
  • Rare pediatric disease and orphan drug designations now obtained in multiple pipeline programs, including TSHA-101 for GM2 gangliosidosis, TSHA-102 for Rett syndrome and TSHA-118 for CLN1

Taysha Gene Therapies Inc. (Nasdaq: TSHA), a patient-centric gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system in both rare and large patient populations, today announced that it has received rare pediatric disease designation and orphan drug designation from the U.S. Food and Drug Administration (FDA) for TSHA-104, an AAV9-based gene therapy in development for SURF1-associated Leigh syndrome. Taysha anticipates it will submit an Investigational New Drug (IND) application to the FDA for TSHA-104 in 2021.

“We have now obtained rare pediatric disease and orphan drug designations in multiple gene therapy programs, which we believe will allow us to work more effectively with the FDA as we advance our broad portfolio,” said RA Session II, President, CEO and Founder of Taysha. “The receipt of these designations highlights the dedication that our team has to advancing our gene therapy pipeline as efficiently and rapidly as possible.”

Leigh syndrome is a severe neurological disorder that usually presents in the first year of life. It is characterized by progressive loss of mental and movement abilities that can result in death within two to three years. Approximately 10-15% of people with Leigh syndrome have a SURF1 mutation.

“Being diagnosed with a mutation in the SURF1 gene is a truly devastating event for families,” said Kasey Woleben, Founder of Cure SURF1 Foundation. “Taysha’s commitment to developing a gene therapy for SURF1 deficiency is greatly welcomed by the patient community and has the potential to save the lives of children afflicted with this progressive disorder.”

Taysha has secured rare pediatric disease designation and orphan drug designation for multiple of its programs, including GM2 gangliosidosis, CLN1, Rett syndrome and now SURF1. In addition to these designations, the company also has fast track status for the CLN1 program.

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10272020

Prevail Therapeutics Receives U.S. FDA Fast Track Designation for PR001 for the Treatment of Neuronopathic Gaucher Disease

Prevail Therapeutics Inc. (Nasdaq: PRVL), a biotechnology company developing potentially disease-modifying AAV-based gene therapies for patients with neurodegenerative diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation for the Company’s experimental gene therapy program, PR001, for the treatment of neuronopathic Gaucher disease (nGD). The Company expects to initiate enrollment of the PROVIDE Phase 1/2 clinical trial of PR001 for Type 2 neuronopathic Gaucher disease patients in the second half of 2020.

“FDA Fast Track designation for PR001 for the treatment of neuronopathic Gaucher disease underscores the significant unmet medical need for this devastating condition, for which there are no currently approved therapies,” said Asa Abeliovich, M.D., Ph.D., Founder and Chief Executive Officer of Prevail. “Type 2 Gaucher disease involves rapidly progressive neurodegeneration leading to death in infancy or early childhood, and Type 3 Gaucher disease is associated with significant neurological manifestations including seizures and motor abnormalities. We believe PR001 has the potential to serve as a much-needed therapeutic option for these patients as enzyme replacement therapies approved for Type 1 Gaucher disease cannot cross the blood brain barrier to address neurological symptoms.”

The FDA previously granted PR001 Rare Pediatric Disease Designation for the treatment of nGD, and Orphan Drug Designation for the treatment of patients with Gaucher disease. In addition, the FDA has granted Fast Track designation for PR001 for the treatment of Parkinson’s disease with GBA1 mutations.

About Fast Track Designation
The FDA’s Fast Track designation is a process designed to expedite or facilitate the review of product candidates to treat serious conditions and fill an unmet medical need. Fast Track designation allows for early and frequent communication with the FDA throughout the entire drug development and review process. It may also allow for priority or rolling review of a company’s Biologics License Application (BLA).

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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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10262020

Aeglea BioTherapeutics Announces U.S. and EU Orphan Drug Designations for ACN00177 for the Treatment of Homocystinuria

Aeglea Biotherapeutics, Inc. (NASDAQ: AGLE), a clinical-stage biotechnology company developing a new generation of human enzyme therapeutics as innovative solutions for rare and other high-burden diseases, today announced the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to ACN00177 for the treatment of Homocystinuria, a serious metabolic disorder characterized by elevated plasma homocysteine which leads to a wide range of life-altering complications and reduced life expectancy. Additionally, the European Medicines Agency (EMA) Committee for Orphan Medicinal Products (COMP) has issued a positive opinion recommending Orphan Drug Designation for ACN00177 for the treatment of Homocystinuria in the European Union. ACN00177 is a novel engineered human enzyme therapy designed to lower the total level of homocysteine in the plasma.

“People with Homocystinuria are living with serious complications, including severe vision issues, skeletal abnormalities, intellectual disability and a high risk of thrombosis which is a common cause of early death, yet many patients do not have effective treatments. These designations underscore the very high unmet need for new treatments for this progressive disease and reaffirms our belief that ACN00177 has the potential to change the lives of people with Homocystinuria,” said Anthony Quinn, M.B Ch.B, Ph.D., president and chief executive officer of Aeglea. “We look forward to dosing the first person in our Phase 1/2 clinical trial of ACN00177 which will move us one step closer to our goal of providing an impactful therapy for people with Homocystinuria who are in need of new treatment options.”

The FDA grants Orphan Drug Designation to drugs and biologics that are intended for the safe and effective treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 people in the U.S. The designation provides certain benefits, including the potential for up to seven years of market exclusivity upon FDA approval, prescription drug user fee waivers and tax credits for qualified clinical trials. The EMA’s Orphan Drug Designation provides benefits, such as protocol assistance, fee reductions and up to ten years of market exclusivity upon regulatory approval, to companies working to develop treatments for life-threatening or chronically debilitating conditions that affect no more than five in 10,000 people in the EU and where no satisfactory treatments are available.

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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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10232020

Protagonist Therapeutics Receives Orphan Drug Designation from the European Medicines Agency for PTG-300 in Polycythemia Vera

Protagonist Therapeutics, Inc. (NASDAQ:PTGX) today announced that the European Medicines Agency (EMA) has granted orphan drug designation for PTG-300 in the treatment of polycythemia vera. PTG-300 is an injectable synthetic peptide mimetic of the natural hormone hepcidin and has previously received orphan drug designation for the treatment of polycythemia vera from the U.S. Food and Drug Administration (FDA).

“This designation reflects the potential of PTG-300 as a treatment candidate and the global need for novel treatments for individuals living with polycythemia vera,” commented Samuel Saks, M.D., Protagonist Chief Medical Officer. “Early clinical results that were reported in May, as well as the novel non-cytoreductive therapeutic mechanism of PTG-300 in regulating iron, suggest that PTG-300 may help a broad population of polycythemia vera patients. We are currently engaged in discussions with leaders in treatment of polycythemia vera to design a pivotal study. Completion of enrollment for the ongoing study of 50 patients is expected in mid-2021. A pivotal study is expected to begin in the second half of 2021, pending our planned discussions with regulatory agencies.”

A Phase 2 study of PTG-300 in patients with polycythemia vera is currently enrolling subjects. Additional information is available at http://ptg300pvstudy.com/.

About Polycythemia Vera

Polycythemia vera is a myeloproliferative neoplasm characterized primarily by the increased production of red blood cells. Well-established treatment guidelines focus on maintaining hematocrit levels continuously below 45 percent to reduce the risk of thrombotic events. Unfortunately, current treatment options are unable to maintain hematocrit to below the 45 percent target for many patients and may be associated with serious side effects. There are an estimated 100,000 patients with polycythemia vera in the U.S. and approximately 100,000 patients in major EU countries.

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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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10212020

FDA grants Glycostem’s oNKord® Orphan Drug Designation for Multiple Myeloma

Glycostem Therapeutics, a leading clinical-stage company focused on the development of therapeutic off-the-shelf Natural Killer (NK) cells, announces it has received the FDA’s Orphan Drug Designation (ODD) for treatment of Multiple Myeloma (MM) patients with its investigational product oNKord®. The designation will provide Glycostem with certain incentives, like eligibility for 7 years of market exclusivity and clear FDA guidance on specific aspects of development for rare diseases. These pave an accelerated path towards market access and treatment of patients suffering from this relatively rare form of cancer.

oNKord® is Glycostem’s first-generation off-the-shelf Natural Killer (NK) cellular immunotherapy product. Over the coming months, AML patients will receive this form of treatment as part of a phase I-IIa (pivotal) trial in AML. A phase II trial for MM patients is expected to start in 2021. This makes Glycostem one of the frontrunners in this promising field of cellular immunotherapy.

“Since 2012 we have been pioneers in the field of developing and manufacturing off-the-shelf Natural Killer cell therapy products for cancer treatment. In 2020 we’re entering a new and exciting phase,” says Troels Jordansen, CEO of Glycostem. “It is great to experience that after receiving FDA and EMA ODD designation for AML, the FDA has also granted us this designation for MM. This allows us to accelerate oNKord®‘s access to the US market and our ultimate ambition: curing cancer.”

Multiple Myeloma (MM) 

MM is the second most common blood cancer, accounting for 15% of blood cancers, and 2% of all cancers. In the US alone it affects more than 130,000 patients; approximately 32,000 Americans are diagnosed with MM each year. MM occurs in infection-fighting plasma cells (a type of white blood cell) found in the bone marrow. These cancerous cells multiply, produce an abnormal protein and push out other healthy blood cells from the bone marrow.

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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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10192020

Versantis Receives FDA Rare Pediatric Disease Designation for VS-01 for the Treatment of Urea Cycle Disorders

Versantis AG, a clinical-stage company developing novel therapies for orphan liver diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted a rare pediatric disease designation (RPDD) to its lead product candidate VS-01, an ammonia clearance enhancer, for the treatment of Urea Cycle Disorders (UCD). UCD is a rare and life-threatening condition caused by an inherited inborn error of metabolism. Current treatment options are associated with poor outcomes.

“The rare pediatric disease designation highlights the potential of using VS-01 to treat the serious and life-threatening manifestations of UCD,” said Meriam Kabbaj, Chief Operations Officer and Co-founder of Versantis. “It is valuable recognition that will help Versantis to ramp up its pediatric program by fostering collaborations with key opinion leader and patients’ associations.”

The FDA grants rare pediatric disease designation for serious or life-threatening diseases primarily affecting children from birth to 18 years and affecting fewer than 200,000 people in the USA. Upon approval of a new drug application, the RPDD renders sponsor companies eligible for a priority review voucher, which can be redeemed to obtain accelerated FDA review of a drug candidate, in any indication, potentially gaining early market access. This voucher may be sold or transferred to another sponsor.

Versantis recently completed a $16M Series B financing round and is currently raising new funds to fuel VS-01 clinical development in rare indications, including UCD.

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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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10162020

Apexigen’s APX005M Granted Orphan Drug Designations for the Treatment of Esophageal and Gastroesophageal Junction Cancer and for the Treatment of Pancreatic Cancer

Apexigen, Inc., a clinical-stage biopharmaceutical company focused on discovering and developing a new generation of antibody therapeutics for oncology, announced today that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation status to APX005M for the treatment of esophageal and gastroesophageal junction cancer and for the treatment of pancreatic cancer.

“These orphan drug designations are important regulatory milestones for Apexigen in its mission to treat cancer patients and for APX005M, which has the potential to meaningfully impact the standard of care across multiple types of solid tumors. We are evaluating APX005M in a broad clinical program that includes more than 10 clinical trials in various indications and therapeutic combinations,” said Xiaodong Yang, MD, PhD, Chief Executive Officer of Apexigen.

The FDA’s Office of Orphan Drug Products grants orphan status to support the development of medicines for underserved patient populations, or rare disorders, that affect fewer than 200,000 people in the United States. Orphan drug designation qualifies the sponsor for various development incentives, including tax credits for qualified clinical testing, up to seven years of marketing exclusivity for the orphan indication and waiver of certain FDA fees.

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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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10152020

Pfizer’s Gene Therapy for Duchenne Placed on Fast Track by FDA

The U.S. Food and Drug Administration (FDA) has granted fast track designation to PF-06939926, Pfizer’s investigational gene therapy for Duchenne muscular dystrophy (DMD).

This designation is given to treatments that show considerable potential in addressing serious conditions for which available treatments fall short. It is meant to speed clinical development, regulatory review — enabling a therapy to qualify for priority review and accelerated approval — and entry into the market upon approval.

“The FDA’s decision to grant our investigational gene therapy PF-06939926 Fast Track designation underscores the urgency to address a significant unmet treatment need for Duchenne muscular dystrophy,” Brenda Cooperstone, MD, Pfizer’s chief development officer for rare disease, said in a press release.

“We are working to advance our planned Phase 3 program as quickly as possible,” she added.

PF-06939926 was previously designated an orphan drug by the FDA and the European Medicines Agency, and given the FDA’s rare pediatric disease designation. These recognitions also support its development and regulatory review, while ensuring marketing exclusivity for a period of time upon approval (seven years in the U.S., 10 years in Europe).

Duchenne is caused by mutations in the DMD gene, resulting in a deficient production of dystrophin, an essential protein for muscle integrity.

To Read the Complete Article at Muscular Dystrophy News, 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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10132020

Allterum Therapeutics granted Orphan Drug Designation and Rare Pediatric Disease Designation for its monoclonal antibody therapy for pediatric leukemia

Allterum Therapeutics, Inc. has been granted both an Orphan Drug Designation and a Rare Pediatric Disease Designation by the FDA for its monoclonal antibody therapy under development for the treatment of pediatric acute lymphoblastic leukemia (ALL). These two designations highlight the medical need and research behind this program and pave the way for an expected Phase I clinical trial in 2021.

Orphan Drug Designation is granted by the FDA to promising new therapies which are being developed for rare indications in the United States. Similarly, Rare Pediatric Disease Designation is granted by the FDA to recognize programs which are specifically geared towards technologies for life-threatening diseases which primarily impact pediatric populations.

While many pediatric patients with ALL respond favorably to standard chemotherapeutics, a subset of patients experience relapsed or refractory disease. In particular, patients with relapsed T-cell ALL have no effective standard therapies available to them. Similarly, additional patients with a subtype of B-cell ALL may not be managed effectively with currently available therapies. Allterum’s antibody therapy is designed to be effective in these patient populations.

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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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10122020

CARsgen Therapeutics Granted Orphan Drug Designation by the US FDA for CT041 CLDN18.2 CAR-T Cells for the Treatment of Gastric and Gastroesophageal Junction Cancers

CARsgen Therapeutics Co., Ltd., a clinical-stage biopharmaceutical company, today announced that the United States (US) Food and Drug Administration (FDA) has granted orphan drug designation to one of CARsgen’s first-in-class drug candidates, CT041, for the treatment of gastric and gastroesophageal junction adenocarcinoma. CT041 is a humanized anti-claudin18.2 autologous chimeric antigen receptor (CAR) T-cell product and is targeted to treat patients with claudin18.2-positive tumors.

CT041 is the first claudin18.2-targeted CAR T-cell therapy that has received Investigational New Drug (IND) clearance by the US FDA and the first to receive IND clearance by the National Medical Products Administration (NMPA) in China. The initiation of an open label, multicenter, Phase 1b clinical trial (NCT04404595) to evaluate the safety and efficacy of autologous CT041 cell therapy in patients with advanced gastric, gastroesophageal, or pancreatic adenocarcinoma is currently underway.

“The orphan drug designation of CT041 by the FDA is of great significance to patients with advanced gastric cancer,” said Dr. Zonghai Li, founder, CEO and CSO of CARsgen. “According to the World Health Organization, about 1,030,000 new cases of gastric adenocarcinoma are expected each year [1]. Despite the development of novel therapies, gastric cancer is still a disease with one of the highest unmet medical needs. Our goal is to continue the development of novel, safe and effective immunotherapies. This is our long-standing commitment to cancer patients worldwide.”

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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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