Tag Archives: orphan drug act

EU Commission granted orphan drug status for SENS-401

Sensorian pharmaceuticals announced that European Commission has granted orphan drug designation in Europe for SENS-401 for the treatment of sudden sensorineural hearing loss. The European Commission grants orphan medicinal product status to provide incentives to develop medicinal products to treat, prevent or diagnose diseases or conditions that affect no more than five in 10,000 persons in the EU. The orphan medicinal product designation provides the company and its drug with certain benefits and incentives in the EU, including a period of market exclusivity of 10 years after the market approval, free consultation and scientific advice on contact with European Medicinal Agency.

 

 

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

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

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

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

Yisheng Biopharma’s Biological Product receives orphan drug status for Hepatocellular Carcinoma

Yisheng Biopharma Co. Ltd. Is a bio pharmaceutical company specialized in development, research, manufacture, marketing and sales of immunotherapeutic products and vaccines, declared that US Food and Drugs Association has granted orphan drug designation to its leading candidate, YS-ON-001 for the treatment of Hepatocellular Carcinoma. YS-ON-001 consist of immune-modulating effects like induction of anti-tumor cytokines, activation of NK cells, regulation of macrophage polarization, and suppression of regulatory T cells.

Previously, YS-ON-001 has acted as a promising agent with anti-tumor efficacy in the treatment of breast, lung, liver and other cancers as compared to the first-line chemotherapies or targeted therapies.

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

CD4CAR therapy receives FDA approval for orphan drug designation for peripheral T-cell Lymphoma

FDA granted orphan drug designation to icell gene therapeutics for its chimeric antigen receptor-engineered T cells directed against the target protein CD4 to treat peripheral T-cell Lymphoma. These chimeric antigens are made up of patient’s own T-cells that are genetically modified to express a protein on its surface.

Action of mechanism of CD4CAR includes by binding to a target protein on another cell and the CAR protein sends a signal to the interior of the T cell to unleash mechanisms that selectively kill the targeted cell.

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

EMA announces orphan drug designation for BP1001 for AML treatment

Bio-Path Holding Inc. declares that European Medicine Agency has granted orphan drug Designation to BP1001 for treatment of acute myeloid leukemia (AML). BP1001 is introduced by Bio-Path Holding Inc. to fill the void of the treatments and medications for effective treatment of acute myeloid leukemia.

In order to get EMA designation of orphan drug, the therapy or treatment must be curing any of the rare life threatening disease with a prevalence of less than 5 in 10,000 people in the European Union. Orphan drug designation provides incentives designed to facilitate development including fee reductions for protocol assistance, scientific advice and importantly, may provide up to ten years of market exclusivity in the EU following product approval.

 

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

FDA announces orphan drug designation for Veliparib as NSCLC treatment

US Food and drug Association (FDA) has declared Veliparib, an oral poly (adenosine diphosphate [ADP]-ribose) polymerase (PARP) inhibitor, developed by Abbvie (ABBV). Currently, Veliparib is undergoing investigation in combination with chemotherapies, such as carboplatin and paclitaxel, or radiation for the treatment of advanced squamous non-small cell lung cancer (NSCLC).

PARP is the naturally occurring enzyme in the body that repairs damage to DNA in the cells. The process is useful to maintain the integrity of healthy cells, and can help in repairing DNA cancer cells, causing them to survive. Veliparib is currently under investigation that its combination with DNA damaging therapies, such as chemotherapy or radiation, may lessen the repair of DNA damage in cancer cells, eventually causing some cells to die.

NSCLC is the common type of lung cancer in the US accounting for 80 to 85% of diagnosed cancer cases. Survival from lung cancer depends on the stage at which cancer is diagnosed.

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