register

News & Trends - Pharmaceuticals

First medicine targeting the root cause of Sickle Cell Disease

Health Industry Hub | November 27, 2019 |

Global Blood Therapeutics announced the approval of a first-in-class therapy in sickle cell disease (SCD) for adults and children 12 years of age and older, by the U.S. Food and Drug Administration (FDA).

Oxbryta (voxelotor), an oral therapy taken once daily, is the first approved treatment that directly inhibits sickle hemoglobin polymerisation, the root cause of SCD.

“This is a major milestone for people living with SCD, their families and those who care for them.” said Ted W. Love, M.D., president and chief executive officer of GBT.

SCD affects millions of people throughout the world. SCD is a lifelong inherited blood disorder that impacts hemoglobin. The sickling process causes haemolytic anaemia and blockages in capillaries and small blood vessels, which impede the flow of blood and oxygen throughout the body. The diminished oxygen delivery to tissues and organs can lead to life-threatening complications, including stroke and irreversible organ damage.5

The accelerated approval of Oxbryta is based on clinically meaningful and statistically significant improvements in hemoglobin levels, accompanied by reductions in red blood cell destruction (haemolysis). Data from the Phase 3 HOPE Study was published recently.

Novartis’ Adakveo (crizanlizumab) was approved recently to reduce frequency of vaso-occlusive crises in sickle cell disease.

Bluebird bio is developing LentiGlobin, a gene therapy for sickle cell disease which is in the early stages of clinical development.

Register FREE to receive the latest news, innovations and insights from Health Industry Hub; the only one-stop-hub connecting Australia’s Pharma, MedTech and Biotech industry professionals and its key stakeholders.

You may also like MSD’s PBS win triples number of lung cancer patients eligible for immuno-oncology therapy


News & Trends - Pharmaceuticals

Federal court dismisses Arrotex dispute against Health Minister

Federal court dismisses Arrotex dispute against Health Minister

Health Industry Hub | July 12, 2024 |

Pharma News: The Federal Court has ruled against Arrotex Pharmaceuticals in their bid to challenge the Minister for Health and […]

More


News & Trends - Pharmaceuticals

Largest diabetes study reveals major benefits of dual therapy with SGLT2 inhibitors and GLP1 receptor agonists

Largest diabetes study reveals major benefits of SGLT2 inhibitors and GLP1 receptor agonists dual therapy

Health Industry Hub | July 12, 2024 |

Pharma News: Combined use of sodium glucose co-transporter 2 inhibitors (SGLT2is) and glucagon-like peptide-1 receptor agonists (GLP1-RAs) is likely to […]

More


News & Trends - Pharmaceuticals

Queensland opens first regional CAR T-cell treatment centre

Queensland launches first regional CAR T-cell treatment centre

Health Industry Hub | July 12, 2024 |

Pharma News: Gilead Sciences and Lymphoma Australia have welcomed the opening of Australia’s first regional CAR T-cell treatment centre at […]

More


News & Trends - Pharmaceuticals

Government inaction leaves essential medicine shortages unresolved

Government inaction leaves essential medicine shortages unresolved

Health Industry Hub | July 12, 2024 |

Pharma News: Over the past 12 months, at least six essential medications and products have been withdrawn from the Australian […]

More


This content is copyright protected. Please subscribe to gain access.