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Complete Response Letter
In United States pharmaceutical regulatory practice, a Complete Response Letter (CRL), or more rarely, a 314.110 letter, is a regulatory action by the Food and Drug Administration in response to a New Drug Application, New Drug Application, Amended New Drug Application or Biologics license application, Biologics License Application, indicating that the application will not be approved in its present form. CRLs replaced Approvable letter, approvable letters in 2018. Background Under the Prescription Drug User Fee Act, the Food and Drug Administration has a limited timespan (known as the PDUFA date) to decide a New Drug Application, New Drug Application, Amended New Drug Application or Biologics license application, Biologics License Application. The FDA may either approve the application or issue a Complete Response Letter. Grounds behind issuing a CRL may include Drug labelling, labelling issues, Good manufacturing practice, current Good Manufacturing Practice concerns or concerns ... [...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), but 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 not d ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Niemann–Pick Disease, Type C
Niemann–Pick type C (NPC) (colloquially, "Childhood Alzheimer's") is a lysosomal storage disease associated with mutations in NPC1 and NPC2 genes. Niemann–Pick type C affects an estimated 1:150,000 people. Approximately 50% of cases present before 10 years of age, but manifestations may first be recognized as late as the sixth decade. Signs and symptoms Niemann–Pick type C has a wide clinical spectrum. Affected individuals may have enlargement of the spleen (splenomegaly) and liver (hepatomegaly), or enlarged spleen or liver combined (hepatosplenomegaly), but this finding may be absent in later onset cases. Prolonged jaundice or elevated bilirubin can present at birth. In some cases, however, enlargement of the spleen or liver does not occur for months or years – or not at all. Enlargement of the spleen or liver frequently becomes less apparent with time, in contrast to the progression of other lysosomal storage diseases such as Niemann–Pick disease, Types A and B or ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Regulation
Regulation is the management of complex systems according to a set of rules and trends. In systems theory, these types of rules exist in various fields of biology and society, but the term has slightly different meanings according to context. For example: * in biology, gene regulation and metabolic regulation allow living organisms to adapt to their environment and maintain homeostasis; * in government, typically regulation means stipulations of the delegated legislation which is drafted by subject-matter experts to enforce primary legislation; * in business, industry self-regulation occurs through self-regulatory organizations and trade associations which allow industries to set and enforce rules with less government involvement; and, * in psychology, self-regulation theory is the study of how individuals regulate their thoughts and behaviors to reach goals. Social Regulation in the social, political, psychological, and economic domains can take many forms: legal restriction ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Trade Secret
Trade secrets are a type of intellectual property that includes formulas, practices, processes, designs, instruments, patterns, or compilations of information that have inherent economic value because they are not generally known or readily ascertainable by others, and which the owner takes reasonable measures to keep secret. Intellectual property law gives the owner of a trade secret the right to restrict others from disclosing it. In some jurisdictions, such secrets are referred to as confidential information. Definition The precise language by which a trade secret is defined varies by jurisdiction, as do the particular types of information that are subject to trade secret protection. Three factors are common to all such definitions: A trade secret is information that * is not generally known to the public; * confers economic benefit on its holder the information is not publicly known; and * where the holder makes reasonable efforts to maintain its secrecy. In internation ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Business Insider
''Insider'', previously named ''Business Insider'' (''BI''), is an American financial and business news website founded in 2007. Since 2015, a majority stake in ''Business Insider''s parent company Insider Inc. has been owned by the German publishing house Axel Springer. It operates several international editions, including one in the United Kingdom. ''Insider'' publishes original reporting and aggregates material from other outlets. , it maintained a liberal policy on the use of anonymous sources. It has also published native advertising and granted sponsors editorial control of its content. The outlet has been nominated for several awards, but is criticized for using factually incorrect clickbait headlines to attract viewership. In 2015, Axel Springer SE acquired 88 percent of the stake in Insider Inc. for $343 million (€306 million), implying a total valuation of $442 million. In February 2021, the brand was renamed simply ''Insider''. History ''Busi ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Valoctocogene Roxaparvovec
Valoctocogene roxaparvovec, sold under the brand name Roctavian, is a gene therapy used for the treatment of hemophilia A. It was developed by BioMarin Pharmaceutical. Valoctocogene roxaparvovec is made of a virus (AAV5) that has been modified to contain the gene for factor VIII, which is lacking in people with hemophilia A. It is given by intravenous infusion. The most common side effects include increased levels of the liver enzymes alanine aminotransferase and aspartate aminotransferase (signs of possible liver problems), increased levels of the enzyme lactate dehydrogenase (sign of possible tissue damage), nausea (feeling sick), and headache. Valoctocogene roxaparvovec was approved for medical use in the European Union in August 2022, and in the United States in June 2023. Medical uses Valoctocogene roxaparvovec is indicated for the treatment of severe haemophilia A (congenital factor VIII deficiency) in adults without a history of factor VIII inhibitors and without dete ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Gene Therapy
Gene therapy is a medical field which focuses on the genetic modification of cells to produce a therapeutic effect or the treatment of disease by repairing or reconstructing defective genetic material. The first attempt at modifying human DNA was performed in 1980, by Martin Cline, but the first successful nuclear gene transfer in humans, approved by the National Institutes of Health, was performed in May 1989. The first therapeutic use of gene transfer as well as the first direct insertion of human DNA into the nuclear genome was performed by French Anderson in a trial starting in September 1990. It is thought to be able to cure many genetic disorders or treat them over time. Between 1989 and December 2018, over 2,900 clinical trials were conducted, with more than half of them in phase I.Gene Therapy Cli ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Haemophilia A
Haemophilia A (or hemophilia A) is a genetic deficiency in clotting factor VIII, which causes increased bleeding and usually affects males. In the majority of cases it is inherited as an X-linked recessive trait, though there are cases which arise from spontaneous mutations. Factor VIII medication may be used to treat and prevent bleeding in people with haemophilia A. Signs and symptoms In terms of the symptoms of haemophilia A, there are internal or external bleeding episodes. Individuals with more severe haemophilia have more severe and more frequent bleeding, while others with mild haemophilia typically have more minor symptoms except after surgery or serious trauma. Moderate haemophiliacs have variable symptoms which manifest along a spectrum between severe and mild forms. Prolonged bleeding from a venepuncture or heelprick is another common early sign of haemophilia, these signs may lead to blood tests which indicate haemophilia. In other people, especially those with modera ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
BioMarin Pharmaceutical
BioMarin Pharmaceutical Inc. is an American biotechnology company headquartered in San Rafael, California. It has offices and facilities in the United States, South America, Asia, and Europe. BioMarin's core business and research is in enzyme replacement therapies (ERTs). BioMarin was the first company to provide therapeutics for mucopolysaccharidosis type I (MPS I), by manufacturing laronidase ( Aldurazyme, commercialized by Genzyme Corporation). BioMarin was also the first company to provide therapeutics for phenylketonuria (PKU). Over the years, BioMarin has been criticised for drug pricing and for specific instances of denying access to drugs in clinical trials. History BioMarin was founded in 1997 by Christopher Starr Ph.D. and Grant W. Denison Jr. with an investment of a $1.5 million from Glyko Biomedical and went public in 1999. Seed investors were amongst others MPM Bioventures, Grosvenor Fund and Florian Schönharting. Business development In 2002, BioMarin acquir ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Arimoclomol
Arimoclomol (INN; originally codenamed BRX-345, which is a citrate salt formulation of BRX-220) is an experimental drug developed by CytRx Corporation, a biopharmaceutical company based in Los Angeles, California. In 2011 the worldwide rights to arimoclomol were bought by Danish biotech company Orphazyme ApS. The European Medicines Agency (EMA) and U.S. Food & Drug Administration (FDA) granted orphan drug designation to arimoclomol as a potential treatment for Niemann-Pick type C in 2014 and 2015 respectively. Mechanism of action Arimoclomol is believed to function by stimulating a normal cellular protein repair pathway through the activation of molecular chaperones. Since damaged proteins, called aggregates, are thought to play a role in many diseases, CytRx believes that arimoclomol could treat a broad range of diseases. Arimoclomol activates the heat shock response. It is believed to act at Hsp70 The 70 kilodalton heat shock proteins (Hsp70s or DnaK) are a family o ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
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