23:12 , May 21, 2019 |  BC Extra  |  Clinical News

BioCryst makes case for oral HAE therapy even as Phase III data disappoint investors

Despite reporting Phase III efficacy data for its oral hereditary angioedema therapy that fell short of marketed injectable therapies to prevent HAE attacks, BioCryst believes the demand for an oral option will enable BCX7353 to...
18:27 , Nov 9, 2018 |  BC Week In Review  |  Clinical News

Adverum reviewing capsid after discontinuing AAT gene therapy

Adverum Biotechnologies Inc. (NASDAQ:ADVM) will discontinue development of gene therapy ADVM-043 to treat α-1 antitrypsin (AAT; A1AT; SERPINA1) deficiency. The company said that although ADVM-043 was well tolerated among six patients in the open-label, U.S....
14:45 , Nov 2, 2018 |  BC Extra  |  Clinical News

Adverum reviewing capsid after discontinuing AAT gene therapy

Adverum Biotechnologies Inc. (NASDAQ:ADVM) sank $1.21 (27%) to $3.34 on Friday after it said it will discontinue development of gene therapy ADVM-043 to treat α-1 antitrypsin (AAT; A1AT; SERPINA1) deficiency. The company said late Thursday...
16:26 , Sep 21, 2018 |  BC Week In Review  |  Clinical News

FDA wants extra trial for Pharming's Ruconest as HAE Prophylactic

Pharming Group N.V. (Euronext:PHARM) said FDA requested another clinical trial of Ruconest conestat alfa as a prophylactic for hereditary angioedema in a complete response letter for an sBLA in the indication. Pharming, which fell €0.20...
18:24 , Sep 19, 2018 |  BC Extra  |  Company News

FDA wants extra trial for Pharming's Ruconest as HAE prophylactic

Pharming Group N.V. (Euronext:PHARM) said FDA requested another clinical trial of Ruconest conestat alfa as a prophylactic for hereditary angioedema in a complete response letter for an sBLA in the indication. Pharming, which fell €0.20...
18:41 , Aug 31, 2018 |  BC Week In Review  |  Clinical News

FDA approves Shire's Takhzyro, first mAb to prevent HAE

Shire plc (LSE:SHP; NASDAQ:SHPG) said FDA approved Takhzyro lanadelumab-flyo to prevent attacks of hereditary angioedema in patients ages 12 and older. The company said Takhzyro is the first mAb approved in the U.S. to prevent...
23:16 , Aug 23, 2018 |  BC Extra  |  Company News

FDA approves Shire’s Takhzyro, first mAb to prevent HAE

Shire plc (LSE:SHP; NASDAQ:SHPG) said FDA approved Takhzyro lanadelumab-flyo to prevent attacks of hereditary angioedema in patients ages 12 and older. The company said Takhzyro is the first mAb approved in the U.S. to prevent...
23:43 , May 31, 2018 |  BC Innovations  |  Emerging Company Profile

Oral route for HAE

The team behind Firazyr icatibant has reunited to develop an oral version of the drug that could give hereditary angioedema patients the alternative to injectables they’ve been asking for. Pharvaris B.V. brings together team members...
19:12 , May 18, 2018 |  BC Week In Review  |  Company News

CSL gains exclusive rights to Cevec's CAPGo technology

Cevec Pharmaceuticals GmbH (Cologne, Germany) granted CSL Ltd. (ASX:CSL) exclusive rights to develop and commercialize CAPGo-derived complement 1 (C1) esterase inhibitor proteins for hereditary angioedema (HAE). CSL plans to develop CAPGo-derived proteins with tailored glycosylation,...
21:02 , Mar 2, 2018 |  BC Week In Review  |  Clinical News

Priority review for Shire's HAE prophylactic

FDA accepted and granted Priority Review to a BLA for lanadelumab (SHP643) from Shire plc (LSE:SHP; NASDAQ:SHPG) to prevent hereditary angioedema (HAE) attacks in patients ages 12 and older. Its PDUFA date is Aug. 26....