Tag Archives: Rare

Albireo’s rare liver disease drug Bylvay scores back-to-back approvals in U.S., Europe

With a pair of approvals on both sides of the Atlantic, liver disease-focused biotech Albireo is off and running with its new medicine Bylvay. The oral drug, the first treatment for pruritus in patients with progressive familial intrahepatic cholestasis (PFIC), scored an FDA nod Tuesday and a European approval Monday. With the FDA’s blessing, Albireo is kicking off an “immediate”… Read More »

FDA to announce new warning on J&J Covid vaccine related to a rare autoimmune disorder, report says

A box of Johnson & Johnson’s Janssen COVID-19 vaccine doses are pictured at Grubb’s Pharmacy on Capitol Hill on Monday, April 12, 2021. Tom Williams | CQ-Roll Call, Inc. | Getty Images The Food and Drug Administration is expected to announce a new warning for Johnson & Johnson‘s Covid-19 vaccine, saying the shot has been… Read More »

Chiesi Global Rare Diseases Announces FDA Approval of Ferriprox (deferiprone) for Treatment of Transfusional Iron Overload due to Sickle Cell Disease

May 1, 2021 Approval is based on demonstrated reduction in liver iron concentration Expanded indications for patients with sickle cell disease or other anemias, as well as thalassemia – BOSTON, May 1, 2021 /PRNewswire/ — Chiesi Global Rare Diseases, a business unit of Chiesi Farmaceutici S.p.A., an international research-focused healthcare Group (Chiesi Group), announced today… Read More »