Monthly Archives: February 2017

SGT-001 declared orphan drug for DMD

Solid Biosciences and Solid GT announces that US Food and Drug Administration (FDA) have granted Orphan drug status to SGT001 for the treatment of Duchenne Muscular Dystrophy (DMD). It is a rare and progressive muscle wasting disease that is usually diagnosed in childhood. SGT001 is the gene therapy mediated to amino-associated viral (AAV) vector. The gene therapy functions by potentially restoring the dystrophin expression in the muscles.

The drug has proved to provide long term systemic expression of comparatively shorter form of dystrophin in the muscles by its clinical studies. The gene therapy also benefits as improved muscle power and prevention from contraction induced damage. The therapy is expected to commercialize in early 2017.

The FDA Orphan Drug Designation is granted to medicines and biologics that are defined as those 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., or that affect more than 200,000 persons but are not expected to recover the costs of developing and marketing a treatment medicine.

 

 

For Orphan Drug Clinical Insight Reports Contact:  neeraj@kuickresearch.com

FDA grants orphan drug status to SER100 in PAH

Serodus announces orphan drug designation for its leading product, SER100 by US Food and Drug Administration (FDA) for the treatment of Pulmonary Arterial Hypertension (PAH). The company is planning for first clinical study in phase IIa or combined phase IIa/III study in patients with PAH.

The FDA Orphan Drug Designation is granted to medicines and biologics that are defined as those 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., or that affect more than 200,000 persons but are not expected to recover the costs of developing and marketing a treatment medicine.

 

 

For Orphan Drug Clinical Insight Reports Contact:  neeraj@kuickresearch.com

Lundbeck’s carnexiv receives FDA orphan drug status

Lundbeck declares orphan drug designation by Food and Drug Association (FDA) for its leading product, Carnexiv injection for the treatment of oral carbamazepine formulations in adults with certain types of seizures when oral administration is temporary not feasible. Carnexiv is the first intravenous formulation of antiepileptic drug (AED) carbamazepine. The pharmaceutical company plans to introduce the drug commercially in early 2017 in the United States.

Carnexiv is a short term alternative therapy for oral carbamazepine formulations that delivers constant care for adult patients who are unable to take the drug by mouth. The oral dosage of the drug consist of certain side effects as dermatologic reactions such as toxic epidermal neurolysis and stevens-johnson syndrome as well as risk of aplastic anemia and agranulocytosis.

The FDA Orphan Drug Designation is granted to medicines and biologics that are defined as those 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., or that affect more than 200,000 persons but are not expected to recover the costs of developing and marketing a treatment medicine.

 

For Orphan Drug Clinical Insight Reports Contact:  neeraj@kuickresearch.com

Eloxx pharmaceuticals receives orphan drug designation for ELX-02 in Mucopolysacchardisis Type 1 (MPS 1) in US

Eloxx Pharmaceuticals, a biotechnology firm focusing on developing and commercializing medicines and treatments for rare diseases, announces that Food and Drug Administration in US and European Medicine Agency (EMA) has granted orphan drug status to its leading product candidate, ELX-02 for the treatment of patients with Mucopolysacchardisis Type 1 (MPS 1).

ELX-02 is a synthetic designer aminoglycoside optimized as a translational read-though drug (TRID) for the treatment of genetic diseases caused by nonsense mutations. The compound is currently under investigation in Phase 1 first in human (FIH) clinical study in healthy volunteers in Israel.

The FDA Orphan Drug Designation is granted to medicines and biologics that are defined as those 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., or that affect more than 200,000 persons but are not expected to recover the costs of developing and marketing a treatment medicine.

The EC decision is based on a recommendation by the European Medicines Agency (EMA) Committee for Orphan Medicinal Products (COMP). Orphan Drug Designation is granted for medicinal products that are intended for the diagnosis, prevention or treatment of rare diseases affecting less than 5 in 10,000 people in the European Union (EU). The Orphan Medicinal Product status of L19TNF entails a number of incentives, including 10-year market exclusivity once the product receives marketing authorisation.

 

For Orphan Drug Clinical Insight Reports Contact:  neeraj@kuickresearch.com

EC grants orphan drug designation to Philogen SpA for the treatment of soft tissue sarcoma.

Philogen SpA, a clinical stage biological company dedicated to develop and commercialize immunology and immune-oncology protein therapeutics, declared that European commission has granted orphan drug designation to L19TNF – a non-covalent trimer of tumor necrosis factor (TNF) fused to an antibody specific to the extra-domain B of fibronectin in single-chain variable fragment format – for the treatment of soft-tissue sarcoma. Soft tissue sarcoma is estimated to affect approximately 2.8 in every 10,000 people in Europe and possess 5year survival rate of 58% for patients suffering from STS.

The EC decision is based on a recommendation by the European Medicines Agency (EMA) Committee for Orphan Medicinal Products (COMP). Orphan Drug Designation is granted for medicinal products that are intended for the diagnosis, prevention or treatment of rare diseases affecting less than 5 in 10,000 people in the European Union (EU). The Orphan Medicinal Product status of L19TNF entails a number of incentives, including 10-year market exclusivity once the product receives marketing authorisation.

 

For Orphan Drug Clinical Insight Reports Contact:  neeraj@kuickresearch.com

 

Invasive Aspergillus infections recieves orphan drug as SCY-078 by SCYNEXIS

Scynexis, a pharmaceutical company specialized in drug development, declared its triterpenoid broad-spectrum antifungal agent, SCY-078 as orphan drug as approved by Food and Drug Administration for the treatment of invasive Aspergillus infections. Previously, the company announced that SCY-078 has received orphan drug designation for the treatment of invasive Candida infections, including candidemia.

 The company was previously granted Qualified Infectious Disease Product (QIDP) designation for both the IV and oral formulations of SCY-078, which provides an additional five years of exclusivity. Together, these designations provide SCYNEXIS with a potential 12 years of market exclusivity in the U.S. upon FDA approval.

The FDA Orphan Drug Designation is granted to medicines and biologics that are defined as those 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., or that affect more than 200,000 persons but are not expected to recover the costs of developing and marketing a treatment medicine.

 

 

For Orphan Drug Clinical Insight Reports Contact:  neeraj@kuickresearch.com

Solid Biosciences receives orphan drug designation from FDA & EMA for Duchenne Muscular Dystrophy

 Solid Bioscience and its subsidiaries declares that Food and Drug Administration and European Medicine Agency has granted orphan drug designation to company’s gene therapy candidate, SGT-001, for the treatment of patients with Duchenne muscular dystrophy (DMD).

Duchenne muscular dystrophy (DMD) is a very rare and progressive muscle damaging disease affecting approximately 1 in 3,500-5,000 boys born worldwide and is the most common fatal genetic disorder diagnosed in childhood. The disease is mainly caused by the absence of dystrophin protein, an important mediator between cardiac and skeletal muscle function. The gene therapy is developed or designed to protect and maintain functional dystrophin expression in the muscles.

The FDA Orphan Drug Designation is granted to medicines and biologics that are defined as those 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., or that affect more than 200,000 persons but are not expected to recover the costs of developing and marketing a treatment medicine.

 

 

For Orphan Drug Clinical Insight Reports Contact:  neeraj@kuickresearch.com

Theranexus’s THN102 receives orphan drug designation from FDA for treatment of Narcolepsy

US Food and Drug Administration has granted orphan drug designation to THN102 developed by Theraneux, a clinical stage biopharmaceutical company, for the treatment of narcolepsy, a rare and highly debilitating sleep/wake disorder. The company has initiated a clinical phase 2 study for determining the potential of THN102 in patients with narcolepsy.

The FDA’s Orphan Drug program offers orphan status to drugs and biologics that are intended for the treatment of rare diseases affecting fewer than 200,000 people in the U.S. The designation provides sponsors with development and commercial incentives for designated compounds and medicines, including eligibility for a seven-year period of market exclusivity in the U.S. after product approval, FDA assistance in clinical trial design and an exemption from FDA user fees.

 

 

For Orphan Drug Clinical Insight Reports Contact:  neeraj@kuickresearch.com

FDA grants orphan drug designation to Synlogic Proprietary Synthetic Biotic

Synlogic, a privately owned bio-pharmaceutical company focusing on developing novel medicines by synthetic biology and microbiome platform, announces that US Food and Drugs Administration grants its investigational program involving a live E. Coli Nissle bacterium modified to assimilate ammonia, for the treatment of urea cycle disorders (UCD). He company accounts for first Live E. Coli Nissle bacterium modified to assimilate ammonia for the potential treatment of UCDs.

UCDs are rare and severe genetic mutation allowing the build-up of toxic levels of ammonia in the bloodstream which causes severe neurological complications and even death. The synlogic’s leading candidate, SYNB1020, is currently undergoing clinical development. The company is planning to file another application for orphan drug status to FDA for its investigational new drug for treatment for UCDs.

The FDA Orphan Drug Designation is granted to medicines and biologics that are defined as those 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., or that affect more than 200,000 persons but are not expected to recover the costs of developing and marketing a treatment medicine.

 

For Orphan Drug Clinical Insight Reports Contact:  neeraj@kuickresearch.com

Phyto-pain pharma declared the submission of orphan drug application for Delta-9-tetrahydrocannibinol and Cannabidiol

PhytoPain Pharma Inc., a subsidiary of GrowPros Cannabis Ventures Inc. stated h submission of two applications for orphan drug designation for Delta-9-tetrahydrocannibinol and Cannabidiol to the US Food and Drugs Administration (FDA), organization responsible for orphan drug development for the treatment of patients with Complex Regional Pain Syndrome Type 1 (CRPS1) and patients with Central Post Stroke Pain (CPSP). The FDA’s decision is still pending, if approved, the company is looking forward for several opportunities and benefits such as most notably tax credits on clinical research reduction of the waiting period and reduced registration fees. It also provides PPP with a 7-year period of market exclusivity in the U.S.

The FDA’s Orphan Drug program offers orphan status to drugs and biologics that are intended for the treatment of rare diseases affecting fewer than 200,000 people in the U.S. The designation provides sponsors with development and commercial incentives for designated compounds and medicines, including eligibility for a seven-year period of market exclusivity in the U.S. after product approval, FDA assistance in clinical trial design and an exemption from FDA user fees.

 

For Orphan Drug Clinical Insight Reports Contact:  neeraj@kuickresearch.com