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Onasemnogene abeparvovec オナセムノジーンアベパルボベック



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Onasemnogene abeparvovec


DNA (synthetic adeno-associated virus 9 vector scAAV9.CB.hSMN human survivor motor neuron protein-specifying)


FDA 2019/5/24 APPROVED

CAS: 1922968-73-7


Spinal muscular atrophy treatment

Treatment of Spinal Muscular Atrophy (SMA) Type 1

Gene therapy product

Image result for Onasemnogene abeparvovec

Onasemnogene abeparvovec, sold under the trade name Zolgensma, is a gene therapy medication used to treat spinal muscular atrophy (SMA).

SMA is a neuromuscular disorder caused by a mutation in the SMN1 gene, which in turn reduces the amount of SMN protein necessary for survival of motor neurons. Onasemnogene abeparvovec is a biologic drug consisting of AAV9 virus capsids that have been deprived of the original viral DNA and instead contain a SMN1 transgene along with promoters. The drug is administered intravenously or intrathecally. Upon administration, the AAV9 viral vector delivers the SMN1 transgene to cell nuclei where the transgene begins encoding SMN protein, thus addressing the root cause of the disease. Since motor neurons do not divide, it is thought that a single dose of the drug will have a lifelong effect.[1]

The medication was developed by a US biotechnology company AveXis, a subsidiary of Novartis,[2] based on an earlier discovery by French researchers.[3] The intravenous formulation was approved in May 2019 in the United States for use in children under 2 years.[4]It carries a list price of US$ 2.125 million per dose (one-time treatment), making it the most expensive medication in the world as of 2019.[5]


Onasemnogene abeparvovec is the international nonproprietary name (INN) and US adopted name (USAN).[6] It was previously known under compound name AVXS-101.

FDA approves a gene therapy that is the most expensive drug in the world

FDA on Friday approved onasemnogene abeparvovec-xioi (Zolgensma—AveXis), a one-time gene therapy for the treatment of spinal muscular atrophy (SMA).

FDA on Friday approved onasemnogene abeparvovec-xioi (Zolgensma—AveXis), a one-time gene therapy for the treatment of spinal muscular atrophy (SMA). The ultrarare disease affects infants. In announcing the approval, Novartis—which acquired AveXis last year—also disclosed the price of the drug, $2.1 million. The company noted that it would provide rebates to insurance companies if the drug is not successful, though it did not offer details about what would be considered failure. Novartis also said it will set up 5-year payment plans for states, small insurance firms, and self-insured employers. Another drug, nusinersen (Spinraza—Biogen) is already available for the treatment of SMA; however, that drug must continue to be injected into patients’ spines throughout their lives, at a cost of $750,000 in the first year and $375,000 a year after that. “Patients with SMA now have another treatment option to minimize the progression of SMA and improve survival,” said Peter Marks, director of FDA’s Center for Biologics Evaluation and Research.


Onasemnogene abeparvovec
Clinical data
Trade names Zolgensma
Synonyms AVXS-101
License data
Routes of
Legal status
Legal status
Pharmacokinetic data
Duration of action lifetime (?)
CAS Number
PubChem CID

Onasemnogene Abeparvovec; AVXS-101; Onasemnogene Abeparvovec [USAN]; DNA (Synthetic Adeno-Associated Virus 9 Vector ScAAV9.CB.HSMN Human Survivor Motor Neuron Protein-Specifying); 1922968-73-7

/////////Onasemnogene abeparvovec, Zolgensma, FDA 2019, オナセムノジーンアベパルボベック ,Spinal muscular atrophy, Gene therapy product, AVXS-101

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DR ANTHONY MELVIN CRASTO, Born in Mumbai in 1964 and graduated from Mumbai University, Completed his Ph.D from ICT, 1991,Matunga, Mumbai, India, in Organic Chemistry, The thesis topic was Synthesis of Novel Pyrethroid Analogues, Currently he is working with GLENMARK LIFE SCIENCES LTD, Research Centre as Principal Scientist, Process Research (bulk actives) at Mahape, Navi Mumbai, India. Total Industry exp 30 plus yrs, Prior to joining Glenmark, he has worked with major multinationals like Hoechst Marion Roussel, now Sanofi, Searle India Ltd, now RPG lifesciences, etc. He has worked with notable scientists like Dr K Nagarajan, Dr Ralph Stapel, Prof S Seshadri, Dr T.V. Radhakrishnan and Dr B. K. Kulkarni, etc, He did custom synthesis for major multinationals in his career like BASF, Novartis, Sanofi, etc., He has worked in Discovery, Natural products, Bulk drugs, Generics, Intermediates, Fine chemicals, Neutraceuticals, GMP, Scaleups, etc, he is now helping millions, has 9 million plus hits on Google on all Organic chemistry websites. His friends call him Open superstar worlddrugtracker. His New Drug Approvals, Green Chemistry International, All about drugs, Eurekamoments, Organic spectroscopy international, etc in organic chemistry are some most read blogs He has hands on experience in initiation and developing novel routes for drug molecules and implementation them on commercial scale over a 30 PLUS year tenure till date June 2021, Around 35 plus products in his career. He has good knowledge of IPM, GMP, Regulatory aspects, he has several International patents published worldwide . He has good proficiency in Technology transfer, Spectroscopy, Stereochemistry, Synthesis, Polymorphism etc., He suffered a paralytic stroke/ Acute Transverse mylitis in Dec 2007 and is 90 %Paralysed, He is bound to a wheelchair, this seems to have injected feul in him to help chemists all around the world, he is more active than before and is pushing boundaries, He has 9 million plus hits on Google, 2.5 lakh plus connections on all networking sites, 90 Lakh plus views on dozen plus blogs, 233 countries, 7 continents, He makes himself available to all, contact him on +91 9323115463, email, Twitter, @amcrasto , He lives and will die for his family, 90% paralysis cannot kill his soul., Notably he has 33 lakh plus views on New Drug Approvals Blog in 233 countries...... , He appreciates the help he gets from one and all, Friends, Family, Glenmark, Readers, Wellwishers, Doctors, Drug authorities, His Contacts, Physiotherapist, etc

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