FDA approves IV artesunate for severe malaria - Regulatory Focus
Posted 28 May 2020 | By
The US Food and Drug Administration (FDA) on Tuesday approved what is now the only treatment for severe malaria in the US following the marketing discontinuation of Eli Lilly’s quinidine in 2019.
The approval was granted to Amivas, LLC for its intravenous (IV) artesunate product, which was co-developed with the US Army under a cooperative research and development agreement (CRADA) that began in 2017 and led to the Army granting Amivas an exclusive license to a patent to manufacture the drug.
While rare in the US, malaria is endemic in other parts of the world, including parts of Central and South America, Africa and Asia. According to the US Centers for Disease Control and Prevention (CDC), most of the 2,000 cases diagnosed in the US each year are acquired during travel and about 300 of those cases are severe.
Outside the US, IV artesunate is widely used as a first line treatment for severe malaria and the drug is included on the World Health Organization’s (WHO) Model List of Essential Medicines.
In an interview with Focus, Amivas Chief Medical Officer Bryan Smith said his company was formed expressly for the purpose of commercializing IV artesunate after earlier efforts by the Army to get the drug approved in the US stalled.
Smith also said he believes the company is eligible for a tropical disease priority review voucher (PRV), which may be redeemed to receive a six-month priority review for another product that otherwise would not be eligible for one. Priority review vouchers may be sold and have often commanded prices in excess of $100 million.
To be eligible for a tropical disease PRV, a product must qualify for a priority review, be intended to treat or prevent a disease on FDA’s list of tropical diseases and cannot contain an active ingredient that has been previously approved by the agency.
The Food and Drug Reauthorization Act of 2017 added a new requirement that applications submitted after 30 September 2017 “must also contain reports of one or more new clinical investigations, (other than bioavailability studies) that are essential to the approval of the application and conducted or sponsored by the sponsor.” The applicant must also attest that the studies were not previously submitted in a marketing application to certain regulators prior to 27 September 2007.
“Amivas did request a priority review voucher and we believe that we have met all the statutory requirements in order for us to be granted one,” Smith said. “It is our understanding that at present, it is still with the FDA and they are still considering the merits of that matter and will revert back to us soon.”
An FDA spokesperson declined to comment on whether the application qualifies for a PRV, citing commercial confidentiality requirements.
In a press release announcing the approval, Smith said that proceeds from the sale of a PRV “have been earmarked to support a US Army nominated Veteran charity, to augment global supply of IV artesunate and to continue postmarketing studies agreed with FDA,” referring to several postmarketing requirement and postmarketing commitment studies specified in the approval letter for IV artesunate.
Since 2007, IV artesunate has been available to US patients through an expanded access investigational new drug (IND) protocol run by the CDC. Through early 2019, when the last remaining quinidine supplies were exhausted, patients only qualified for IV artesunate via the expanded access protocol if they could not access quinidine or were intolerant of quinidine, or if it was contraindicated or ineffective.
“This created delays and also frustration and confusion in clinicians in the United States,” Smith said. “As a fully available commercial product, there will no longer be those burdens or hurdles and there will no longer be the additional paperwork, consent process and reporting processes that are necessary to maintain an open treatment IND.”
For the time being, IV artesunate will remain available through the CDC’s expanded access protocol until Amivas is able to build out its commercial distribution network.
Amivas has not said how much the drug will cost once it is available, but Smith said believes, “The value of the product will be reflected in the price,” and noted the complexity and challenge present in supplying an orphan drug nationwide that requires rapid administration to be effective.
“This approval will now give patients more access to a lifesaving drug,” said John Farley, acting director of the Office of Infectious Diseases at FDA’s Center for Drug Evaluation and Research.
The approval was granted to Amivas, LLC for its intravenous (IV) artesunate product, which was co-developed with the US Army under a cooperative research and development agreement (CRADA) that began in 2017 and led to the Army granting Amivas an exclusive license to a patent to manufacture the drug.
While rare in the US, malaria is endemic in other parts of the world, including parts of Central and South America, Africa and Asia. According to the US Centers for Disease Control and Prevention (CDC), most of the 2,000 cases diagnosed in the US each year are acquired during travel and about 300 of those cases are severe.
Outside the US, IV artesunate is widely used as a first line treatment for severe malaria and the drug is included on the World Health Organization’s (WHO) Model List of Essential Medicines.
In an interview with Focus, Amivas Chief Medical Officer Bryan Smith said his company was formed expressly for the purpose of commercializing IV artesunate after earlier efforts by the Army to get the drug approved in the US stalled.
Smith also said he believes the company is eligible for a tropical disease priority review voucher (PRV), which may be redeemed to receive a six-month priority review for another product that otherwise would not be eligible for one. Priority review vouchers may be sold and have often commanded prices in excess of $100 million.
To be eligible for a tropical disease PRV, a product must qualify for a priority review, be intended to treat or prevent a disease on FDA’s list of tropical diseases and cannot contain an active ingredient that has been previously approved by the agency.
The Food and Drug Reauthorization Act of 2017 added a new requirement that applications submitted after 30 September 2017 “must also contain reports of one or more new clinical investigations, (other than bioavailability studies) that are essential to the approval of the application and conducted or sponsored by the sponsor.” The applicant must also attest that the studies were not previously submitted in a marketing application to certain regulators prior to 27 September 2007.
“Amivas did request a priority review voucher and we believe that we have met all the statutory requirements in order for us to be granted one,” Smith said. “It is our understanding that at present, it is still with the FDA and they are still considering the merits of that matter and will revert back to us soon.”
An FDA spokesperson declined to comment on whether the application qualifies for a PRV, citing commercial confidentiality requirements.
In a press release announcing the approval, Smith said that proceeds from the sale of a PRV “have been earmarked to support a US Army nominated Veteran charity, to augment global supply of IV artesunate and to continue postmarketing studies agreed with FDA,” referring to several postmarketing requirement and postmarketing commitment studies specified in the approval letter for IV artesunate.
Since 2007, IV artesunate has been available to US patients through an expanded access investigational new drug (IND) protocol run by the CDC. Through early 2019, when the last remaining quinidine supplies were exhausted, patients only qualified for IV artesunate via the expanded access protocol if they could not access quinidine or were intolerant of quinidine, or if it was contraindicated or ineffective.
“This created delays and also frustration and confusion in clinicians in the United States,” Smith said. “As a fully available commercial product, there will no longer be those burdens or hurdles and there will no longer be the additional paperwork, consent process and reporting processes that are necessary to maintain an open treatment IND.”
For the time being, IV artesunate will remain available through the CDC’s expanded access protocol until Amivas is able to build out its commercial distribution network.
Amivas has not said how much the drug will cost once it is available, but Smith said believes, “The value of the product will be reflected in the price,” and noted the complexity and challenge present in supplying an orphan drug nationwide that requires rapid administration to be effective.
“This approval will now give patients more access to a lifesaving drug,” said John Farley, acting director of the Office of Infectious Diseases at FDA’s Center for Drug Evaluation and Research.
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