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Priority Review
Priority review is a program of the United States Food and Drug Administration (FDA) to expedite the review process for drugs that are expected to have a particularly great impact on the treatment of a disease. The priority review voucher program is a program that grants a voucher for priority review to a drug developer as an incentive to develop treatments for disease indications with limited profitability. Priority review vouchers are currently earned by pharmaceutical companies for the development and approval of drugs treating neglected tropical diseases, rare pediatric diseases, and "medical countermeasures" for terrorism. The voucher can be used for future drugs that could have wider indications for use, but the company is required to pay a fee (approximately $2.8 million) to use the voucher. When seeking approval for a drug, manufacturers can apply to the FDA for priority review. This is granted when a drug is intended to treat a serious condition and would "provide a sig ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Food And Drug Administration
The United States Food and Drug Administration (FDA or US FDA) is a List of United States federal agencies, federal agency of the United States Department of Health and Human Services, Department of Health and Human Services. The FDA is responsible for protecting and promoting public health through the control and supervision of food safety, tobacco products, caffeine products, dietary supplements, Prescription drug, prescription and Over-the-counter drug, over-the-counter pharmaceutical drugs (medications), vaccines, biopharmaceuticals, blood transfusions, medical devices, electromagnetic radiation emitting devices (ERED), cosmetics, Animal feed, animal foods & feed and Veterinary medicine, veterinary products. The FDA's primary focus is enforcement of the Federal Food, Drug, and Cosmetic Act (FD&C). However, the agency also enforces other laws, notably Section 361 of the Public Health Service Act as well as associated regulations. Much of this regulatory-enforcement work is ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Miltefosine
Miltefosine, sold under the trade name Impavido among others, is a medication mainly used to treat leishmaniasis and free-living amoeba infections such as '' Naegleria fowleri'' and '' Balamuthia mandrillaris''. This includes the three forms of leishmaniasis: cutaneous, visceral and mucosal. It may be used with liposomal amphotericin B or paromomycin. It is taken by mouth. Common side effects include vomiting, abdominal pain, fever, headaches, and decreased kidney function. More severe side effects may include Stevens–Johnson syndrome or low blood platelets. Use during pregnancy appears to cause harm to the baby and use during breastfeeding is not recommended. How it works is not entirely clear. Miltefosine was first made in the early 1980s and studied as a treatment for cancer. A few years later it was found to be useful for leishmaniasis and was approved for this use in 2002 in India. It is on the World Health Organization's List of Essential Medicines. Medical uses L ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Sebelipase Alfa
Sebelipase alfa, sold under the brand name Kanuma, is a recombinant form of the enzyme lysosomal acid lipase (LAL) that is used as a medication for the treatment of lysosomal acid lipase deficiency (LAL-D). Text was copied from this source which is copyright European Medicines Agency. Reproduction is authorized provided the source is acknowledged. It is administered via intraveneous infusion. It was approved for medical use in the European Union and in the United States in 2015. Medical uses Sebelipase alfa is indicated for long-term enzyme replacement therapy (ERT) in people of all ages with lysosomal acid lipase (LAL) deficiency. History Sebelipase was developed by Synageva that became part of Alexion Pharmaceuticals in 2015. For its production, chickens are genetically modified to produce the recombinant form of LAL (rhLAL) in their egg white. After extraction and purification it becomes available as the medication. On 8 December 2015 the FDA announced that its appr ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Alexion Pharmaceuticals
Alexion Pharmaceuticals, Inc., a subsidiary of AstraZeneca, is a pharmaceutical company headquartered in Boston, Massachusetts that specializes in orphan drugs to treat rare diseases. Its products include eculizumab (Soliris) and ravulizumab (Ultomiris), both used to treat the rare disorders of atypical hemolytic uremic syndrome (aHUS) and paroxysmal nocturnal hemoglobinuria (PNH); asfotase alfa (Strensiq), used to treat hypophosphatasia; sebelipase alfa (Kanuma), used to treat lysosomal acid lipase deficiency, and andexanet alfa (Andexxa), used to stop life threatening or uncontrollable bleeding in people who are taking rivaroxaban or apixaban. With costs that can reach as much as $2 million per year, the drugs manufactured by Alexion are some of the most expensive drugs worldwide. History Alexion Pharmaceuticals was founded in 1992 at Science Park in New Haven, Connecticut by Steven Squinto and Leonard Bell, a physician at Yale New Haven Hospital and assistant profess ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Strensiq
Asfotase alfa, sold under the brand name Strensiq, is a medication used in the treatment of people with perinatal/infantile- and juvenile-onset hypophosphatasia. U.S. Patent 7,763,712 The most common side effects include injection site reactions, hypersensitivity reactions (such as difficulty breathing, nausea, dizziness and fever), lipodystrophy (a loss of fat tissue resulting in an indentation in the skin or a thickening of fat tissue resulting in a lump under the skin) at the injection site, and ectopic calcifications of the eyes and kidney. The enzyme tissue non-specific |
Orotic Aciduria
Orotic aciduria (AKA hereditary orotic aciduria) is a disease caused by an enzyme deficiency, resulting in a decreased ability to synthesize pyrimidines. It was the first described enzyme deficiency of the ''de novo'' pyrimidine synthesis pathway. Orotic aciduria patients lack the enzyme known as UMP synthase. Orotic aciduria is characterized by excessive excretion of orotic acid in urine because of the inability to convert orotic acid to UMP. It causes megaloblastic anemia and may be associated with mental and physical developmental delays. Orotic aciduria is a rare disease. Fewer than 30 cases in human history have been reported in literature. Signs and symptoms Patients typically present with excessive orotic acid in the urine, failure to thrive, developmental delay, and megaloblastic anemia which cannot be cured by administration of vitamin B12 or folic acid. Cause This autosomal recessive disorder is caused by a deficiency in the enzyme UMPS, a bifunctional protein th ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Xuriden
Uridine triacetate ( INN), formerly known as vistonuridine, is an orally active tri- acetylated prodrug of uridine used: * in the treatment of hereditary orotic aciduria (brand name Xuriden ); * to treat people following an overdose of chemotherapy drugs 5-fluorouracil (5-FU) or capecitabine regardless of the presence of symptoms, or who exhibit early-onset, severe or life-threatening toxicity affecting the cardiac or central nervous system, and/or early-onset, unusually severe adverse reactions (e.g., gastrointestinal toxicity and/or neutropenia) within 96 hours following the end of fluorouracil or capecitabine administration (brand name Vistogard). Uridine triacetate was developed, manufactured and distributed by Wellstat Therapeutics. It was granted breakthrough therapy designation by the U.S. Food and Drug Administration The United States Food and Drug Administration (FDA or US FDA) is a List of United States federal agencies, federal agency of the United States D ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Asklepion Pharmaceuticals
An Asclepieion ( ''Asklepieion''; Ἀσκλαπιεῖον in Doric dialect; Latin ''aesculapīum''), plurally Asclepieia, was a healing temple in ancient Greece (and in the wider Hellenistic and Roman world) that was dedicated to Asclepius, the first doctor-demigod in Greek mythology. Asclepius was said to have been such a skilled doctor that he could even raise people from the dead. Stemming from the myth of his great healing powers, pilgrims would flock to temples built in his honor in order to seek spiritual and physical healing. Asclepieia included carefully controlled spaces conducive to healing and fulfilled several of the requirements of institutions created for healing. Treatment at these temples largely centered around promoting healthy lifestyles, with a particular emphasis on a person's spiritual needs. Characteristic of the Asclepieion was the practice of '' incubatio'', also known as 'temple sleep.' This was a process by which patients would go to sleep in the tem ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Zellweger Syndrome
Zellweger syndrome is a rare congenital disorder characterized by the reduction or absence of functional peroxisomes in the cells of an individual. It is one of a family of disorders called Zellweger spectrum disorders which are leukodystrophy, leukodystrophies. Zellweger syndrome is named after Hans Zellweger (1909–1990), a Swiss-American pediatrician, a professor of pediatrics and genetics at the University of Iowa who researched this Disease, disorder. Signs and symptoms Zellweger syndrome is one of three peroxisome biogenesis disorders that belong to the Zellweger spectrum of peroxisome biogenesis disorders (PBD-ZSD). The other two disorders are neonatal adrenoleukodystrophy (NALD), and infantile Refsum disease (IRD). Although all have a similar molecular basis for disease, Zellweger syndrome is the most severe of these three disorders. Zellweger syndrome is associated with impaired neuronal migration, neuronal positioning, and brain development. In addition, individuals ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Cholic Acid
Cholic acid, also known as 3α,7α,12α-trihydroxy-5β-cholan-24-oic acid is a primary bile acid that is insoluble in water (soluble in alcohol and acetic acid), it is a white crystalline substance. Salts of cholic acid are called cholates. Cholic acid, along with chenodeoxycholic acid, is one of the two major bile acids produced by the liver, where it is synthesized from cholesterol. These two major bile acids are roughly equal in concentration in humans. Derivatives are made from cholyl-CoA, which exchanges its CoA with either glycine, or taurine, yielding glycocholic and taurocholic acid, respectively. Cholic acid downregulates cholesterol-7-α-hydroxylase (rate-limiting step in bile acid synthesis), and cholesterol does the opposite. This is why chenodeoxycholic acid, and not cholic acid, can be used to treat gallstones (because decreasing bile acid synthesis would supersaturate the stones even more). Cholic acid and chenodeoxycholic acid are the most important huma ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
United Therapeutics
United Therapeutics Corporation is an American biotechnology company that develops pharmaceuticals and technologies related to organ transplantation, including xenotransplantation. Many of the company's products are focused towards lung disease and organ manufacturing. United Therapeutics is co-headquartered in Silver Spring, Maryland and Research Triangle Park, North Carolina, with additional facilities in Magog and Bromont, Quebec; Melbourne and Jacksonville, Florida; Blacksburg, Virginia; and Manchester, New Hampshire. History United Therapeutics was founded in 1996 by Martine Rothblatt, an American lawyer, author, and entrepreneur, who created Sirius XM. In 1994, Rothblatt's young daughter was diagnosed with a fatal orphan disease, pulmonary arterial hypertension (PAH). Rothblatt sold her telecom stock and started the PPH Cure Foundation to fund PAH research. In 2002, the U.S. Food and Drug Administration (FDA) approved United Therapeutics' drug Remodulin, a prostacycl ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
