| |   | Good morning! Amylyx just secured a Canadian approval for its ALS drug... we'll see if U.S. regulators follow. CRISPR-based therapy for sickle cell disease and beta-thalassemia seems to have legs, and more.
- Meghana | | | Amylyx's ALS drug approved by Health Canada Canadian regulators have approved a new drug for amyotrophic lateral sclerosis from Amylyx Pharmaceuticals. The drug, called Albrioza, is still under FDA scrutiny, and an approval decision is expected in September, STAT’s Adam Feuerstein writes. The data underpinning this approval come from a single study that shows the drug significantly slowed disease progression. Health Canada approved Albrioza “with conditions,” meaning it will need confirmatory studies after the drug hits the market. Results are expected in 2024. The drug will be available to Canadians with ALS in about six weeks, after Amylyx lays out the drug’s price and negotiates with payers. Read more. | How did San Diego become a biotech hub, and will it last? San Diego may not quite match Boston or San Francisco, but it’s a booming biotech hub in its own right — and has been for some four decades, when Hybritech, the region’s first life sciences company, began churning out custom antibodies en masse. Today, the city’s biotech community is vast, with dozens of life sciences startups working busily in the shadow of giants like Illumina. This week, the Biotechnology Innovation Organization’s international convention is being held in San Diego — which means the time’s ripe to delve into the colorful history of medical innovation in the region. STAT’s Jonathan Wosen dives deep into SoCal’s flourishing — but wildly expensive — biotech sector. Read more. | July 26 | A conversation with Admiral Rachel Levine Admiral Levine has used her position to prioritize the challenges that members of the LGBTQ+ community face when trying to access health care. Join her as she’ll take the virtual stage as part of our STATUS List Spotlight series. Register now. | A CRISPR therapy as a functional cure? CRISPR-based therapy for patients with sickle cell disease and beta-thalassemia is showing prolonged benefit, new data show. The therapy, administered as a one-time infusion, was developed jointly by Vertex Pharmaceuticals and CRISPR Therapeutics. All 31 patients given the treatments reported being free of severe pain; 42 of 44 patients with beta-thalassemia no longer required blood transfusions. These are considered functional cures for these inherited diseases — even though not even half of the patients studied have been followed for at least a year, which is considered the minimum benchmark for confirming a drug’s benefit. “I have seen first-hand the impact that this investigational therapy has had on patients in these clinical trials and continue to be impressed by the totality of the data,” said a physician who presented the data Sunday at the annual European Hematology Association meeting. Read more. | A step forward in vaccinating children Both Pfizer's and Moderna’s Covid-19 vaccines are safe and effective for young children, FDA regulators said in briefing documents released ahead of an advisory committee meeting to decide whether to authorize the shots. Moderna’s vaccine was 51% effective at preventing symptomatic infection in children 6 months to 2 years old, and 37% effective in children 2 to 5 years old. Pfizer and its partner BioNTech had previously said their vaccine was about 80% effective in preventing symptomatic Covid-19 among children ages 6 months through 4 years. | More reads - Simmering disputes persist as Covid patent waiver talks come down to the wire. STAT
- FDA advisors endorse Bluebird gene therapy for rare blood disorder. STAT
- GSK says RSV vaccine for older adults ‘offers exceptional protection’ STAT
- Listen: Diverse clinical trials: Why aren’t we there yet? STAT
| Thanks for reading! Until tomorrow,  | | | |
No comments