The use of the United States FDA programs as a strategy to advance the development of drug products for neglected tropical diseases

Sachs‐Barrable, Kristina; Conway, Jocelyn; Gershkovich, Pavel; Ibrahim, Fady

doi:10.3109/03639045.2014.884132

Cited by 17 publications

(10 citation statements)

References 7 publications

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“…The tropical disease voucher was initially developed to incentivize companies for working on treatments for selected tropical diseases in 2007 (9). The Rare Pediatric Disease Priority Review Voucher was created under the Food and Drug Administration Safety and Innovation Act (FDASIA) and was based on this tropical disease voucher (10).…”

Section: Vouchers As Incentivesmentioning

confidence: 99%

Incentives for Starting Small Companies Focused on Rare and Neglected Diseases

Ekins

Wood

2015

Pharm Res

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Starting biotech or pharmaceutical companies is traditionally thought to be based around a scientist, their technology platform or a clinical candidate spun out from another company. Between us we have taken a different approach and formed two small early stage companies after initially leveraging the perspective of a parent with a child with a life-threatening rare disease. Phoenix Nest (http://www.phoenixnestbiotech.com/) was co-founded to work on treatments for Sanfilippo syndrome a devastating neurodegenerative lysosomal storage disorder. In the space of just over 3 years we have built up collaborations with leading scientists in academia and industry and been awarded multiple NIH small business grants. The second company, Collaborations Pharmaceuticals Inc. (http://www.collaborationspharma.com/) was founded to address some of the other 7000 or so rare diseases as well as neglected infectious diseases. The Rare Pediatric Disease Priority Review Voucher is likely the most important incentive for companies working on rare diseases with very small populations. This may also be partially responsible for the recent acquisitions of rare disease companies with late stage candidates. Lessons learned in the process of starting our companies are that rare disease parents or patients can readily partner with a scientist and fund research through NIH grants rather than venture capital or angel investors initially. This process may be slow so patience and perseverance is key. We would encourage other pharmaceutical scientists to meet rare disease parents, patients or advocates and work with them to further the science on their diseases and create a source of future drugs.

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Section: Vouchers As Incentivesmentioning

confidence: 99%

Incentives for Starting Small Companies Focused on Rare and Neglected Diseases

Ekins

Wood

2015

Pharm Res

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“…While the definition of NTDs varies, this category generally includes diseases that impact marginalized populations or lack effective treatment or vaccine. Sometimes, the products that are available to treat a NTD, such as the NTD schistosomiasis, are not easily accessible to the affected populations . Schistosomiasis, caused by trematode flatworms of the genus Schistosoma , is one of the most significant NTDs in the world.…”

Section: Introductionmentioning

confidence: 99%

“…Neglected tropical diseases (NTDs) are a group of biologically unrelated tropical diseases that disproportionally affect low-income populations in developing regions of Africa, Asia, and the Americas. [1] While the definition of NTDs varies, this category generally includes diseases that impact marginalized populations or lack effective treatment or vaccine. Sometimes, the products that are available to treat a NTD, such as the NTD schistosomiasis, are not easily accessible to the affected populations.…”

Section: Introductionmentioning

confidence: 99%

“…Sometimes, the products that are available to treat a NTD, such as the NTD schistosomiasis, are not easily accessible to the affected populations. [1] Schistosomiasis, caused by trematode flatworms of the genus Schistosoma, is one of the most significant NTDs in the world. Despite great advances in its treatment and prevention, this disease is still significantly prevalent, has high morbidity rates, and affects more than 240 million people worldwide.…”

Section: Introductionmentioning

confidence: 99%

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Schistosomicidal Activity of Dihydrobenzofuran Neolignans

Dias

Patrocinio

Pagotti

et al. 2018

Chemistry & Biodiversity

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We have evaluated the antischistosomal activity of synthetic dihydrobenzofuran neolignans (DBNs) derived from (±)-trans-dehydrodicoumaric acid dimethyl ester (1) and (±)-trans-dehydrodiferulic acid dimethyl ester (2) against adult Schistosoma mansoni worms in vitro. Compound 4 ((±)-trans-4-O-acetyldehydrodiferulic acid dimethyl ester) displayed the most promising activity; at 200 μm, it kills 100 ± 0% of worms after 24 h, which resembles the result achieved with praziquantel (positive control) at 1.56 μm. The hydrogenation of the double bond between C7' and C8', the introduction of an additional methyl group at C3', and a double bond between C7 and C8 decreased the schistosomicidal activity of DBNs. On the other hand, the presence of the acetoxy group at C4 played an interesting role in this activity. These results demonstrated the interesting schistosomicidal potential of DBNs, which could be further exploited.

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“…time during which the originator can market the drug without competition from generic drug manufacturers) by the number of months that the review period is shortened. The effectiveness of this approach and the rules under which a PRV is awarded have been examined critically (Sonderholm, 2009, Robertson et al., 2012, Sachs-Barrable et al., 2014, Kesselheim et al., 2015). The ’case’ of miltefosine for treatment of visceral leishmaniasis has pointed attention to the fact that a PRV can be awarded to an organisation which has not itself invested significant funds in the development and for a drug which has already been registered in another country for the neglected disease indication.…”

Section: Introductionmentioning

confidence: 99%

Research for new drugs for elimination of onchocerciasis in Africa

Kuesel

2016

International Journal for Parasitology: Drugs and Drug Resistan

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Onchocerciasis is a parasitic, vector borne disease caused by the filarial nematode Onchocerca volvulus. More than 99% of the population at risk of infection live in Africa. Onchocerciasis control was initiated in West Africa in 1974 with vector control, later complemented by ivermectin mass drug administration and in the other African endemic countries in 1995 with annual community directed treatment with ivermectin (CDTI.) This has significantly reduced infection prevalence. Together with proof-of-concept for onchocerciasis elimination with annual CDTI from foci in Senegal and Mali, this has resulted in targeting onchocerciasis elimination in selected African countries by 2020 and in 80% of African countries by 2025. The challenges for meeting these targets include the number of endemic countries where conflict has delayed or interrupted control programmes, cross-border foci, potential emergence of parasite strains with low susceptibility to ivermectin and co-endemicity of loiasis, another parasitic vector borne disease, which slows down or prohibits CDTI implementation. Some of these challenges could be addressed with new drugs or drug combinations with a higher effect on Onchocerca volvulus than ivermectin. This paper reviews the path from discovery of new compounds to their qualification for large scale use and the support regulatory authorities provide for development of drugs for neglected tropical diseases. The status of research for new drugs or treatment regimens for onchocerciasis along the path to regulatory approval and qualification for large scale use is reviewed. This research includes new regimens and combinations of ivermectin and albendazole, antibiotics targeting the O. volvulus endosymbiont Wolbachia, flubendazole, moxidectin and emodepside and discovery of new compounds.

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The use of the United States FDA programs as a strategy to advance the development of drug products for neglected tropical diseases

Cited by 17 publications

References 7 publications

Incentives for Starting Small Companies Focused on Rare and Neglected Diseases

Incentives for Starting Small Companies Focused on Rare and Neglected Diseases

Schistosomicidal Activity of Dihydrobenzofuran Neolignans

Research for new drugs for elimination of onchocerciasis in Africa

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