Marisa Wexler MS,  —

Marisa holds an MS in Cellular and Molecular Pathology from the University of Pittsburgh, where she studied novel genetic drivers of ovarian cancer. She specializes in cancer biology, immunology, and genetics. Marisa began working with BioNews in 2018, and has written about science and health for SelfHacked and the Genetics Society of America. She also writes/composes musicals and coaches the University of Pittsburgh fencing club.

Articles by Marisa Wexler

Soliris safely used during pregnancy for two women with PNH

Soliris (eculizumab) was successfully used to help manage paroxysmal nocturnal hemoglobinuria (PNH) during a pregnancy in two women. Based on observations made in these cases, the scientists noted that Soliris “seems to be generally safe in pregnancy for both mother and fetus,” though they emphasized a need for additional…

FDA approves Voydeya as add-on therapy for adults with PNH

The U.S. Food and Drug Administration (FDA) has approved Voydeya (danicopan) as an add-on treatment for extravascular hemolysis, or the destruction of red blood cells taking place outside blood vessels, in adults with paroxysmal nocturnal hemoglobinuria (PNH). The treatment is to be taken with the standard-of-care therapies Ultomiris…

PNH treatment Fabhalta recommended for EU approval

A European Medicines Agency committee has recommended that Novartis’ twice-daily oral therapy Fabhalta (iptacopan) be approved for the treatment of paroxysmal nocturnal hemoglobinuria (PNH). The recommendation from the Committee for Medicinal Products for Human Use (CHMP) supports Fabhalta’s use as a monotherapy, or stand-alone treatment, in adults with…

PNH treatment ruxoprubart granted FDA orphan drug status

The U.S. Food and Drug Administration (FDA) has granted orphan drug status to ruxoprubart, an experimental treatment for paroxysmal nocturnal hemoglobinuria (PNH) that’s now in Phase 2 clinical testing. The drug is designed to control the genetic disease while posing a lower infection risk than currently available treatments. Orphan…

Danicopan, now Voydeya, approved in Japan as add-on PNH treatment

Danicopan has been approved in Japan as a first-in-class, add-on therapy for people with paroxysmal nocturnal hemoglobinuria (PNH) who have an insufficient response to treatment with C5 inhibitors. Its developer Alexion, AstraZeneca Rare Disease will be marketing it under the brand name Voydeya, the company announced in a…

Phase 2 trial of NM8074 in untreated PNH patients underway

NovelMed Therapeutics has launched a Phase 2 clinical trial to test its experimental therapy, NM8074, in people with paroxysmal nocturnal hemoglobinuria (PNH) who haven’t received treatment for the disease. Eight patients have been enrolled so far and two have started treatment, according to the company. The Phase…

Phase 1 trial of oral BCX10013 enrolling PNH patients in S. Africa

A proof-of-concept clinical trial of the experimental oral therapy BCX10013 is enrolling people with paroxysmal nocturnal hemoglobinuria (PNH) in Cape Town, South Africa, with plans to expand to other sites where approved PNH treatments are not available. The Phase 1 study (NCT06100900) is open to adults with PNH…

Use of cemdisiran may allow for reduced Soliris dosing in PNH

The experimental treatment cemdisiran (ALN-CC5) failed to fully control hemolysis, or red blood cell destruction, in a small clinical trial involving people with paroxysmal nocturnal hemoglobinuria (PNH). However, the therapy’s use was found to reduce the dose of Soliris (eculizumab) needed to keep blood cell destruction under control…