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Mashup Score: 1FDA Approves Aflibercept for Treating Retinopathy of Prematurity - 1 year(s) ago
This the fifth indication approved for aflibercept but the first approved orphan drug indication.d
Source: CheckRareCategories: Hem/Onc News and Journals, Latest HeadlinesTweet
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Mashup Score: 0FDA Approves Omaveloxolone to Treat Friedreich’s Ataxia - 1 year(s) ago
Omaveloxolone activates nuclear factor erythroid 2-related factor 2 (NrF2), a transcription factor that is impaired in patients with Friedreich’s ataxia.
Source: CheckRareCategories: Hem/Onc News and Journals, Latest HeadlinesTweet-
The US Food and Drug Administration has approved omaveloxolone #Skyclarys to treat persons 16 years and older with Friedreich’s ataxia @US_FDA @Friedreichsatax #checkrare #rarediseases #FDA #orphandrug https://t.co/uXNC1t9Q9E
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Mashup Score: 1FDA Approves Aflibercept for Treating Retinopathy of Prematurity - 1 year(s) ago
This the fifth indication approved for aflibercept but the first approved orphan drug indication.d
Source: CheckRareCategories: Hem/Onc News and Journals, Latest HeadlinesTweet-
The US Food and Drug Administration #FDA has approved aflibercept #Eylea to treat preterm infants with retinopathy of prematurity. @US_FDA #checkrare #raredisease #orphandrug https://t.co/kM08wEvUCQ
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Mashup Score: 0Ocugen Announces OCU400 Receives Orphan Drug Designations for Retinitis Pigmentosa and Leber Congenital Amaurosis - Eyewire+ - 1 year(s) ago
Ocugen announced that the FDA granted orphan drug designations to OCU400—human nuclear hormone receptor subfamily 2 group E member 3 (hNR2E
Source: Eyewire+Categories: Latest Headlines, OphthalmologyTweet-
.@Ocugen announces FDA grants #orphandrug designations to OCU400—human nuclear hormone receptor subfamily 2 group E member 3 (hNR2E3)—for the treatment of #retinitispigmentosa (RP) and Leber congenital amaurosis (LCA). https://t.co/mjMnYjfnuv https://t.co/NSgcUOlp2i
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Mashup Score: 2Regulators Keep Rare Diseases Open For Business - 2 year(s) ago
The cost of rare diseases to patients and society at large is 10 times higher than for many of the biggest mass market diseases combined. With 95% of rare diseases lacking a treatment or cure, there is an economic imperative for policymakers to keep encouraging pharmaceutical companies to develop more orphan drug…
Source: In VivoCategories: Latest Headlines, Rare DiseaseTweet-
"It’s clear that the patient community understands how important the existing #orphandrug exclusivity interpretation is in driving #research and giving them hope for a treatment.” - NORD's Heidi Ross in a recent @INVIVOnow article. Read more: https://t.co/tkbjA10Tpx
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Mashup Score: 4'No magic bullet': For drugmakers and the FDA, clinical trials on ultra-rare diseases pose thorny challenges - 2 year(s) ago
“You either accept the uncertainties around rare disease approvals — that a drug is not going to have the same kind of safety data as others — or not,” said the FDA’s Norman Stockbridge.
Source: STATCategories: General Medicine News, Latest HeadlinesTweet-
‘No magic bullet’: For drugmakers and the FDA, clinical trials on ultra-rare diseases pose thorny challenges.. https://t.co/DUyu6O0o7Q #pharma #biotech #FDA #RareDisease #orphandrug $MITO $AGLE $RARE
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Mashup Score: 2
Efanescotocog alfa is the first factor VIII therapy to be granted Breakthrough Therapy designation by the FDA.
Source: HCPLive®Categories: Latest Headlines, Rare DiseaseTweet-
Efanescotocog alfa is the first factor VIII therapy to be granted Breakthrough Therapy designation by the #FDA. #HemophiliaA #OrphanDrug https://t.co/fxxIrBUNZl
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Mashup Score: 0
“Vague laws can be exploited, resulting in fewer patients getting the treatments they need to survive,” said Sen. Bill Cassidy.
Source: STATCategories: General Medicine News, Latest HeadlinesTweet-
Lawmakers wade into a contentious battle over orphan drug monopolies.. https://t.co/w5pPOYPqhn #pharma #monopoly #raredisease #orphandrug #FDA $CPRX
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Mashup Score: 0Unintended Consequences: Balancing innovation, profiteering within the Orphan Drug Act - 3 year(s) ago
The Orphan Drug Act, passed in good faith to help patients with rare diseases in 1983, has been at the center of multiple controversies in recent years. A study published last month in Health Affairs has added fuel to that fire.The key issue is that drugs granted orphan status may be given market exclusivity, allowing for lucrative monopolies and no competition. Some manufacturers have used this
Source: www.healio.comCategories: Latest Headlines, RheumatologyTweet-
Although the #OrphanDrug Act has brought relief for #RareDisease patients, balancing its incentives against possible abuses of market exclusivity has become a complicated issue https://t.co/OGdY2S7GZz
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Mashup Score: 0Unintended Consequences: Balancing innovation, profiteering within the Orphan Drug Act - 3 year(s) ago
The Orphan Drug Act, passed in good faith to help patients with rare diseases in 1983, has been at the center of multiple controversies in recent years. A study published last month in Health Affairs has added fuel to that fire.The key issue is that drugs granted orphan status may be given market exclusivity, allowing for lucrative monopolies and no competition. Some manufacturers have used this
Source: www.healio.comCategories: Latest Headlines, RheumatologyTweet-
Since it was passed in 1983, the #OrphanDrug Act has struggled to balance incentives for #RareDisease drug development against #BigPharma profiteering https://t.co/7Z0vSJ78va https://t.co/up86MhQ1rH
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The US Food and Drug Administration #FDA has approved aflibercept #Eylea to treat preterm infants with retinopathy of prematurity. @US_FDA #checkrare #raredisease #orphandrug https://t.co/kM08wEvUCQ