Tag Archives: orphan drug market act

FDA grants orphan drug status to Keystone’s ceramides

Keystone Nano has announced that its leading product, ceramides has received orphan drug status from U.S Food and Drug Administration (FDA) for the treatment of patients with liver cancer. Demonstration of ceramides have proved its effective use in killing cancer cells while leaving normal cells alone in a human body and proving treatment without any toxicity.

The pharmaceutical agency, Keystone Nano has designed a unique NanoLiposome which helps in delivering ceramides to the cancer cells in the patient’s body. Liver cancer is estimated to cause death in approximately 25,000 people in United States and 700,000 people across the globe. Currently, the life threatening disease does not on any effective treatment option in the market.

Orphan Drug designation is granted to those medications or therapy that are rare, more specifically, affect less than 200,000 patients in the country. These rare diseases are incurable and severe in nature and may cause death. To fulfill the need of treat options for individuals, FDA grants orphan drug designation to drugs which are investigated to be helpful in the treatment of such rare diseases. Orphan drug status is a package of several financial benefits for the drugs and its manufacturers such as certain tax credits and exemption from paying prescription drug user fees normally required from companies submitting products for approval.



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

European Union grants orphan drug status to Crenolanib

Arog Pharmaceuticals declared that its leading drug candidate has received orphan drug designation for the treatment of patients with acute myeloid leukemia and soft tissue sarcoma.

 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



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

FDA grants orphan drug designation to AAV1-FS344 for Inclusion Body Myositis

US Food and Drug Association (FDA) announces orphan drug designation for Milo Biotechnology’s AAV1-FS344 for treatment of inclusion body myositis. AAV1-FS344 is a gene therapy delivered follistatin protein that increases muscle strength and function.

Currently, AAV1-FS344 is in its phase I/II trial in the Nationwide Children’s Hospital in adult patients with sporadic inclusion body myositis which is funded by the foundations Parent Project Muscular Dystrophy and The Myositis Association.

Inclusion body myositis (IBM) is an adult onset myopathy causing severe and progressive muscle weakness. Most of the patients suffering from IBM are wheelchair bound within 10 years of diagnosis. Milo’s AAV1-FS344 program is also being studied for other two rare diseases namely, Becker muscular dystrophy and Duchenne muscular dystrophy, for which the company received FDA approved orphan drug status in 2012.

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

MediciNova declares FDA approval for MN-166 as orphan drug

Medicinova, a biopharmaceutical company, announced FDA approved orphan drug designation for its leading medical treatment, MN-166 (ibudilast) for the treatment of Amyotrophic Lateral Sclerosis (ALS). Currently, there is only one approved treatment for ALS that is riluzole, present in the market. MN-166 is presently undergoing clinical trials by researchers at Carolinas HealthCare System’s (CHS).

Amyotrophic Lateral Sclerosis (ALS) is also called as Lou Gehrig’s disease is a progressive neuro-degenerative disease that affects brain and the spinal cord. ALS drains the ability to trigger specific muscles causing weakness in them. It affects the voluntary movement in the patient and causes total paralysis at later stages.

Orphan Drug designation is granted by the FDA’s Office of Orphan Products Development for drugs that are expected to provide significant therapeutic advantage over existing treatments and that target conditions affecting 200,000 or fewer U.S. patients annually. Orphan Drug designation qualifies a company for several benefits under the Orphan Drug Act 1983. These benefits include assistance with clinical study design in drug development, tax credits for qualified clinical trials costs, exemptions from certain FDA application fees, and seven years of market exclusivity upon regulatory product approval.



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