REZLIDHIA (olutasidenib), FDA-Approved for Treatment of Adult Patients with Relapsed or Refractory Acute Myeloid Leukemia, Available at Biologics by McKesson
CARY, N.C., Dec. 19, 2022 — Biologics by McKesson, an independent specialty pharmacy specializing in oncology and rare disease areas, has been selected by Rigel Pharmaceuticals as one of two specialty pharmacies in a limited distribution network for REZLIDHIATM (olutasidenib), which is indicated for... (Source: McKesson News)
Source: McKesson News - December 19, 2022 Category: Information Technology Source Type: news
KRAZATI ® (adagrasib), FDA Approved for Treatment of Advanced Non-Small Cell Lung Cancer Harboring the KRASG12C Mutation, Available at Biologics by McKesson
CARY, N.C., Dec. 14, 2022 — Biologics by McKesson, an independent specialty pharmacy focused on oncology and rare disease areas, has been selected by Mirati therapeutics as one of two specialty pharmacies in a limited distribution network for KRAZATI® (adagrasib), which is indicated for the treatmen... (Source: McKesson News)
Source: McKesson News - December 14, 2022 Category: Information Technology Source Type: news
Accelerating Rare disease Cures (ARC) Program
The mission of CDER ’s ARC Program is to drive scientific and regulatory innovation and engagement to accelerate the availability of treatments for patients with rare diseases. (Source: FDA Center for Drug Evaluation and Research - What's New)
Source: FDA Center for Drug Evaluation and Research - What's New - December 13, 2022 Category: Drugs & Pharmacology Authors: FDA Source Type: news
Amgen dives deeper into rare disease drugs with $27.8 bln Horizon deal
Dec 12 (Reuters) - Amgen Inc (AMGN.O) on Monday agreed to buy Horizon Therapeutics Plc (HZNP.O) in a deal valued at $27.8 billion, fortifying its rare diseases portfolio in the biggest buyout in the sector this year. The company will pay $116.50 in cash, a premium of nearly 20% to the stock's last…#refinitiv #chemocentryxinc #medicare #williamblair #bankofamerica #horizontherapeuticsplc #trimbleinc #coupasoftwareinc #coupasoftware #enbrel (Source: Reuters: Health)
Source: Reuters: Health - December 13, 2022 Category: Consumer Health News Source Type: news
Amgen/Horizon: swoop on rare disease specialist will push up debt load
The strategic logic of buying Horizon is hard to fault. But good assets do not come cheap (Source: FT.com - Drugs and Healthcare)
Source: FT.com - Drugs and Healthcare - December 12, 2022 Category: Pharmaceuticals Source Type: news
Biotech Leader Shares How Rocket Pharmaceuticals Is Working To Eradicate Rare Diseases In Children
COO and President of Rocket Pharmaceuticals, Kinnari Patel ’s, shares how the company is using gene therapy to eradicate diseases in children. (Source: Forbes.com Healthcare News)
Source: Forbes.com Healthcare News - December 12, 2022 Category: Pharmaceuticals Authors: Yola Robert, Senior Contributor Tags: ForbesWomen /forbeswomen Leadership /leadership Innovation /innovation Healthcare /healthcare Source Type: news
Amgen to buy Horizon Therapeutics in $26.4B deal
Amgen is spending more than $26 billion to dive deeper into rare disease treatments with a deal for drugmaker Horizon Therapeutics (Source: ABC News: Health)
Source: ABC News: Health - December 12, 2022 Category: Consumer Health News Tags: Health Source Type: news
Three things to know about the accelerated approval pathway
If you or a loved one were to receive a diagnosis of a serious or life-threatening disease, you would likely want access to a U.S. Food and Drug Administration (FDA) approved safe and effective medicine as soon as possible. That ’s precisely why the accelerated approval pathway was created back in 1992 at the urging of patient advocates. Since then, it has been a lifeline for millions of patients. Primarily utilized for cancers, rare diseases and HIV/AIDS, the pathway allows patients earlier access to potentially lifesavi ng medicines – often when no other treatment is available and when patients are facing difficult o...
Source: The Catalyst - December 12, 2022 Category: Pharmaceuticals Authors: Lucy Vereshchagina Tags: FDA Prescription Drug Safety Source Type: news
ELAHERE ™ (mirvetuximab soravtansine-gynx), FDA Approved for Platinum-Resistant Ovarian Cancer, Available at Biologics by McKesson
CARY, N.C., Dec. 12, 2022 — Biologics by McKesson, an independent specialty pharmacy specializing in oncology and rare disease areas, has been selected by ImmunoGen, Inc., a leader in the expanding field of antibody-drug conjugates (ADCs), as the sole specialty pharmacy provider for ELAHERE™ (mirvet... (Source: McKesson News)
Source: McKesson News - December 12, 2022 Category: Information Technology Source Type: news
Millions Suffer From Untreatable Rare Diseases While Medical Costs...
New studies highlight the devastating economic burden of rare diseases. Impacting about 30 million people in the United States alone, the direct and indirect medical costs of rare diseases approach $1...(PRWeb December 12, 2022)Read the full story at https://www.prweb.com/releases/millions_suffer_from_untreatable_rare_diseases_while_medical_costs_skyrocket/prweb19069084.htm (Source: PRWeb: Medical Pharmaceuticals)
Source: PRWeb: Medical Pharmaceuticals - December 12, 2022 Category: Pharmaceuticals Source Type: news
We doctors must improve our approach to rare diseases | Letter
The mantra of ‘hear hooves, think horse not zebra’ is greatly contributing to diagnostic delays and poor patient experience, saysDr Lucy McKayThank you for highlighting the Hughes ’s story about having a family member with an undiagnosed condition (‘Nobody knows what’s wrong with me’ – life with an undiagnosed condition, 6 December). The stories are all too familiar to me and motivate my work every day. I am a second-generation rare disease advocate. My brother died of a rare genetic condition before I was born. My mum founded a charity to connect families and drive research on this condition back in the 1980...
Source: Guardian Unlimited Science - December 11, 2022 Category: Science Authors: Guardian Staff Tags: Health Medicine Medical research Doctors NHS Science Education Society Genetics Source Type: news
Roche ’s subcutaneous crovalimab given every four weeks achieves disease control in people with PNH, a life-threatening blood condition
The phase III COMMODORE 3 study ofcrovalimab met primary endpoints of transfusion avoidance andhaemolysis control in people with paroxysmal nocturnal hemoglobinuria (PNH)1COMMODORE 3 is the first China-specific study in PNH. Current treatment options are extremely limited in China, resulting in significant levels of disease-related morbidity and mortality for people living with PNH2Based on these datacrovalimab has received Breakthrough Therapy Designation and is under Priority Review for approval in ChinaBasel, 11 December 2022 - Roche (SIX: RO, ROG; OTCQX: RHHBY) today announced positive new data from the phase III COMMO...
Source: Roche Media News - December 11, 2022 Category: Pharmaceuticals Source Type: news
Roche ’s subcutaneous crovalimab given every four weeks achieves disease control in people with PNH, a life-threatening blood condition
The phase III COMMODORE 3 study ofcrovalimab met primary endpoints of transfusion avoidance andhaemolysis control in people with paroxysmal nocturnal hemoglobinuria (PNH)1COMMODORE 3 is the first China-specific study in PNH. Current treatment options are extremely limited in China, resulting in significant levels of disease-related morbidity and mortality for people living with PNH2Based on these datacrovalimab has received Breakthrough Therapy Designation and is under Priority Review for approval in ChinaBasel, 11 December 2022 - Roche (SIX: RO, ROG; OTCQX: RHHBY) today announced positive new data from the phase III COMMO...
Source: Roche Investor Update - December 11, 2022 Category: Pharmaceuticals Source Type: news
What It ’s Like to Live With Stiff Person Syndrome
twIn an emotional social-media video posted Dec. 8, singer Celine Dion informed fans that she has been diagnosed with a rare neurological disease called stiff person syndrome. A diagnosis wasn’t easy or straightforward. “I’ve been dealing with problems with my health for a long time…we now know this is what’s been causing all of the spasms that I’ve been having,” said Dion, who is 54.
Here’s what to know about the condition and what it feels like.
What is stiff person syndrome?
According to the National Institute of Neurological Disorders and Stroke, stiff person syndrome has...
Source: TIME: Health - December 9, 2022 Category: Consumer Health News Authors: Alice Park Tags: Uncategorized Disease healthscienceclimate Source Type: news
Supreme Court dismisses petition seeking removal of GST, customs duty on medicines to cure spinal muscular atrophy
Cure SMA Foundation of India, a parent-led organisation, had approached the court seeking exemption of GST and customs duty on treatment of patients suffering from the disease. However, the court said, “Ultimately it is for the government to take a policy decision whether to completely exempt any drug for treatment of rare diseases from levy of IGST, CGST, SGST or customs duty. No writ of mandamus can be issued for directing the Union to exempt drugs from payment of tax or duty.” (Source: The Economic Times Healthcare and Biotech News)
Source: The Economic Times Healthcare and Biotech News - December 9, 2022 Category: Pharmaceuticals Source Type: news
