Tag Archives: global rare disease

EU Union grants orphan drug status to GLPG1690 for idiopathic pulmonary fibrosis

Galapagos, a bio-technology company dedicated to produce small molecule medicines with novel modes of action, declared that its novel medication GLPG1690 has been approved as orphan drug by European Commission for the treatment of idiopathic pulmonary fibrosis (IPF).

IPF is a chronic fibrotic disorder of lungs affecting people above the age of 40 years. IPF is a rare disease as its prevalence has been estimated to be 30 per 100,000 people in Europe as well as US. Presently, there are no medications available to cure IPF.

The FDA and EMA’s designation of orphan status is granted to encourage new developments and therapies for rare diseases and disorders. Orphan status can be granted to those diseases that are rare. These incentives include protocol assistance, i.e. scientific advice specific for designated orphan medicines, and 10 years of market exclusivity once the medicine is on the market.

 

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Firdapse gets orphan drug status for treatment of Myasthenia Gravis

Catalyst pharmaceuticals Inc., a biopharmaceutical company, declare its novel therapy Firdapse (amifampridine phosphate) as orphan drug for the treatment of patients with Myasthenia Gravis, as approved by Food and Drug Association (FDA). The company focuses on developing and commercializing new therapies for the treatment of rare debilitating diseases.

Myasthenia Gravis is the rare neuromuscular disease antibodies to the muscle-specific kinase (MuSK-MG) which inflicts approximately 5-8% of Myasthenia Gravis patients which is equal to 4,500 patients in the US. The characteristics of myasthenia gravis are predominance in women, prominent bulbar involvement, sever clinical conditions and resistant to treatment. Presently, there are anticholinesterase inhibitors or immunosuppressant available for the treatment of the disease.

FDA provides a number of benefits to the drug developers or pharmaceutical companies through development and commercialization. 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

EMA declares orphan drug designation to Masitinib for Lou Gehrig’s disease

AB Science SA, a pharmaceutical company specialized in research, development and commercialization of protein kinase inhibitors (PKIs), announces that European Medicines Agency has granted Masitinib, orphan drug designation  for the treatment of Amyotrophic Lateral Sclerosis (ALS), also known as Lou Gehrig’s disease.

Masitinib is orally transmitted tyrosine kinase inhibitor that attacks the most important immune cells namely macrophages and mast cells. With its unique method of inhibition, it helps curing diseases related to central nervous system and inflammatory diseases.

The guidelines and procedure to apply for orphan drug status is slightly different from that of Food and Drug Association. Under EMA rules, in situations where there already exist authorized medicinal products, the sponsor should provide justification for the assumption that the medicinal product for which designation is sought will be of significant benefit to those affected by the condition.

Secondly, the application being based on an assumption of significant benefit, a comparison with authorized treatments is required. Thirdly, to follow the spirit of the Orphan legislation, which makes it clear that an orphan application may be made at any stage of the development, ‘significant benefit’ will be based on the available evidence at the stage of designation.

 

 

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

First orphan drug introduced for Friedreich’s Ataxia: FDA

Agilis Biotherapeutics, LLC (Agilis), a biotechnology company dedicated to innovation of DNA therapies for rare genetic diseases damaging the central nervous system, declared that Food and Drug Association (FDA)and United States Food has granted orphan drug designation to AGIL-FA , company’s gene therapy product candidate to treat patients of Friedreich’s Ataxia.

Friedreich’s Ataxia (FA) is an inherited degenerative neuromuscular disorder resulting in loss of motor coordination and strength, hearing, vision, speech and often premature death. It is primarily caused by a defect in the FXN gene that lowers the production of the frataxin protein.

Agilis gene therapy is considered to be the first one in the list of FDA approved therapies for treatment of FA. Also the company holds fourth position in the list of all orphan designated drugs declared by FDA this year. Agilis gene is designed to deliver corrective DNA to specific CNS cells to restore frataxin protein levels in patient’s body.

FDA grants orphan drug status for rare and life threatening diseases that affect less than 200,000 people per year in the U.S. Also, FDA provide several beneficiaries to drug developers including assistance with clinical study design and 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

TGA grants orphan drug status to Raxone for DMD

Santhera Pharmaceuticals ‘s lead drug candidate, Raxone, has been approved by Therapeutic Goods Administration (TGA) as an orphan drug to treat patients with Duchenne Muscular Dystrophy (DMD). Raxone, under the name of Catena, has already received same status by the European, Swiss, and U.S regulatory authorities.

Raxone has been designed to show effective results in patients with disfunctioning of respiratory organ. The company describes it as a synthetic short-chain benzoquinone and a cofactor for the enzyme NAD(P)H: quinone oxidoreductase (NQO1). The drug is made to stimulate mitochondrial electron transport, reducing and scavenging reactive oxygen species (ROS or free radicals) and supplementing cellular energy that which are highly dependent on mitochondrial activity.

TGA provides investigational treatments for rare disorders that affect less than 2000 people in that country. Also, the drug qualifies for market exclusivity post approval of the designation.

 

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

First orphan drug approved for Duchenne muscular dystrophy

Food and drug Association (FDA), U.S, has announced Exondys 51 (eteplirsen) injection as the orphan drug for treatment of patients with Duchenne muscular dystrophy (DMD). Exondys 51 has been developed by Sarepta Therapeutics of Cambridge, Massachusetts. It is specifically indicated for patients who have a confirmed mutation of the dystrophin gene amenable to exon 51 skipping, which affects about 13 percent of the population with DMD.

DMD is an uncommon genetic disorder characterized by progressive muscle deterioration and paleness. Absence of dystrophin, a protein generally helps in tightening muscle cell. DMD is found to be most common kind of muscular dystrophy. Its occurrence is recorded to be one in every 3,600 male infants worldwide and rare cases in females. The disease causes severe weakness in patients in their early teens followed by life-threatening heart and respiratory conditions during their adulthood.

Exondys 51’s fast track approval is based on the surrogate endpoint of dystrophin increase in skeletal muscle observed in some Exondys 51-treated patients. The most common side effects reported by patients taking Exondys 51 in the clinical trials were balance disorder and vomiting.

FDA grants orphan drug status for rare and life threatening diseases that affect less than 200,000 people per year in the U.S. Also, FDA provide several beneficiaries to drug developers including assistance with clinical study design and 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

FDA declares Firdapse as orphan drug for treatment of Myasthenia Gravis

Catalyst Pharmaceuticals, a company dedicated to drug discovering and developing for patients with rare diseases, announces that orphan drug designation has been granted to Firdapse to treat patients suffering from myasthenia gravis, by Food and Drug Association (FDA).

Antibodies to the muscle-specific kinase (MuSK-MG), results in a rare Myasthenia Gravis disease, found to be dominant in females and characterized by prominent bulbar involvement, more severe clinical condition and resistance to treatment. Many patients with Myasthenia Gravis are recorded to be unresponsive to the current treatment with anticholinesterase inhibitors or immunosuppressant.

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. The benefits apply across all stages of drug development and include an accelerated approval process; seven years of market exclusivity following marketing approval; tax credits on U.S. clinical trials; eligibility for Orphan Drug grants; and waiver of Prescription Drug User Fee Act (PDUFA) and certain other administrative fees.

 

 

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

 

FDA grants orphan drug designation to BIV201 for treatment of Acsites

BioVie Inc., a company dedicated to discovering and developing therapeutic drugs, declares that it’s new drug candidate BIV201 has received orphan drug designation for the treatment of acsites due to chronic liver cirrhosis.

Currently, BioVie is looking forward to complete an investigational new drug (IND) application for BIV201 having potent vasoconstrictor as an active ingredient, which qualifies the drug for effective treatment of various life threatening diseases including type 1 hepatorenal syndrome (HRS), esophageal variceal bleeds, and sepsis.

 

 

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

Coversin by Akari therapeutics receives Orphan drug Designation from FDA

Coversin developed by Akari therapeutics, an emerging pharmaceuticals company dedicated to the development and commercialization of innovative therapeutics to treat orphan autoimmune and inflammatory diseases has been granted orphan drug designation for the treatment of Paroxysmal Nocturnal Hemoglobinuria.

PNH is an ultra-rare, life-threatening and debilitating disease of the blood with estimated 8,000-10,000 patients across North America and Europe. The PNH patients suffer from a certain genetic deficiency in which they allow their own complement system to attack and destroy blood cells, leading to life-threatening complications.

According to FDA, orphan drug designation status is given to those drugs or treatments which promise to cure or prevent rare life threatening diseases. By FDA, rare diseases are defined as those which affect less than 200,000 people in US. Also, FDA helps the companies which cultivate such drugs by providing them with incentives to sponsor developing drugs and biologics. The receipt of Orphan Drug Designation status does not change the regulatory requirements or process for obtaining marketing approval and designation does not mean that marketing approval will be received.

 

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

EMA grants BioMarin’s Brinuera orphan drug status for CLN2 disease

BioMarin pharmaceuticals Inc. declares its leading drug candidate, Brinuera as orphan drug as approved by European Medicinal Agency (EMA) for the treatment of a form of Batten disease, CLN2 disease usually found in children. The disease is generally diagnosed in children of 2 to 4 years of age causing them to lose their ability to walk by 6 years of age and the patient may die while reaching to 12 years of age.. Symptoms of CLN2 include language delay and seizures, followed by movement disorders, motor deterioration, dementia and blindness.

The European medicinal Agency (EMA) 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.

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