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 »

CDC panel criticized for postponed meeting on COVID-19 vaccines, rare heart issues

An advisory panel to the Centers for Disease Control and Prevention drew criticism after it postponed a discussion on heart inflammation among a small fraction of COVID-19 vaccine recipients in observance of the Juneteenth National Independence Day holiday.  The Advisory Committee on Immunization Practices’ (ACIP) initially scheduled meeting on Friday, June 18 will now be… 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 »

Johnson & Johnson’s COVID-19 vaccine wins renewed backing from U.S. experts after probe into rare blood clots

After officials halted Johnson & Johnson’s COVID-19 vaccine rollout in its tracks last week, U.S. vaccine advisers agreed on Friday that vaccinations should resume. Going forward, the shot will carry a warning about rare and severe blood clots, they said. Of the nearly 8 million doses administered, officials identified 15 cases of thrombosis with thrombocytopenia (TTS), CDC vaccine advisers said Friday. Three vaccine recipients who developed TTS have died. All… Read More »