Jean-Jacques Bienaimé (BioMarin via Youtube)
The FDA on Friday signed off on an accelerated approval for Biomarin’s Voxzogo (vosoritide) injection, the first treatment to target the underlying genetics of dwarfism, which can increase the height of children five years of age and older with the condition. The injection, first approved in Europe with a $300,000 annual price tag, works by binding to a specific receptor called natriuretic peptide receptor-B, which reduces the growth regulation gene’s activity and stimulates bone growth in children.
‘Today’s approval fulfills an unmet medical need for more than 10,000 children in the United States,’ said Theresa Kehoe, director of FDA’s Division of General Endocrinology.
The approval is based on a Phase III placebo-controlled trial in which 121 participants were randomly assigned to receive either Voxzogo injections under the skin or a placebo.
‘Researchers measured the participants’ annualized growth velocity, or rate of height growth, at the end of the year. Participants who received Voxzogo grew an average 1.57 centimeters taller compared to those who received a placebo,’ the FDA said.
A condition of the accelerated approval is that BioMarin must run a post-marketing study that will assess the final adult height.
Jean-Jacques Bienaimé, chairman and CEO of BioMarin, said in a statement, ‘Voxzogo is a medical first that is rooted in BioMarin’s focus on molecular genetics and targets the underlying cause of the condition. More than a decade of scientific research underpins the medical advance that Voxzogo represents. We thank the FDA for recognizing its value as the first therapeutic treatment option for children with achondroplasia.’
But there’s an ethical question at the heart of the approval too. According to Stat News, organizations such as Little People of America have long sought to advocate for fairness while noting that achondroplasia, the most common form of dwarfism, doesn’t preclude them from a fulfilling life, and drugs like Voxzogo could be a threat to the community they’ve built.
For years, paper-based processes and individual point solutions dominated the clinical research landscape, and patient participation in clinical trials was largely an in-person engagement. But when the COVID-19 pandemic took a stronghold, traditional clinical trial methods emerged as inadequate, putting clinical trials and the life sciences industry at a crossroads. Practically overnight, the industry had to rapidly shift to decentralized clinical trial methods, while maintaining data quality and regulatory compliance.
Douglas Fambrough, Dicerna CEO (Dicerna via YouTube)
Early this year researchers at Novo Nordisk were beaming as they announced the first drug identified in their RNAi alliance with Dicerna was headed into the clinic. And now they’re coming back for the whole thing.
This morning the Copenhagen-based pharma giant put out word that it is buying Dicerna $DRNA — an RNAi pioneer that has had its up and downs over the years — for $3.3 billion. Novo is paying $38.25 a share — an 80% premium.
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The European Medicines Agency on Friday said that Merck’s antiviral molnupiravir can now be used to treat adults with Covid-19 who do not require supplemental oxygen and who are at increased risk of developing severe disease.
The pill, known commercially in Europe as Lagevrio, has not yet been authorized in the US and has to be taken twice a day for 5 days. The EMA said it should be administered as soon as possible after diagnosis of Covid, and within 5 days of the start of symptoms. The quick authorization is based on data showing the drug reduced the chance that a newly diagnosed Covid-19 patient would be hospitalized or die by 50%, according to data presented by the company in October.
Last week, Bristol Myers Squibb marched out long-term data for its heart drug mavacamten ahead of what execs had hoped would be a positive FDA decision in January. But regulators are saying they need a bit more time to think.
The FDA has extended mavacamten’s PDUFA date three months, from Jan. 28 to April 28, Bristol Myers announced on Friday. The news came just a few days after independent drug pricing watchdog ICER raised concerns about the candidate’s long-term safety in its final evidence report.
Peter Marks (Jim Lo Scalzo/Pool via AP Images)
All US adults who received two doses of an mRNA Covid-19 vaccine are now eligible for a booster shot, the FDA announced Friday.
The moves will amend the existing EUAs for the Pfizer/BioNTech and Moderna boosters, which had previously been limited to immunocompromised individuals, those older than 65 and adults at high risk for severe disease through certain comorbidities or occupational exposure. For Moderna in particular, the booster shots come in a half-dose of the original vaccine series — 50 µg instead of 100 µg — while Pfizer’s remains at 30 µg for each shot.
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Gilead is going all in — hook, line and sinker — on its oncology alliance with Arcus. And they are going for broke.
The big biotech unveiled a deal that now delivers $725 million in opt-in payments covering the clinical development programs for Arcus, ranging from their closely watched anti-TIGIT programs for domvanalimab and AB308 to etrumadenant (the A2a/A2b adenosine receptor antagonist) and quemliclustat, the small molecule CD73 inhibitor. Gilead will also cover half of the development costs, handing Terry Rosen’s biotech a deal that gives them a clear cash runway to achieving all its goals in oncology.
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Michel Vounatsos (Biogen via YouTube)
The FDA tells Endpoints News today that it’s aware of the death of a patient taking Biogen’s controversial Alzheimer’s drug Aduhelm (aducanumab) and is ‘actively investigating’ the case.
Their statement follows the latest comments from RBC analyst Brian Abrahams, who is back with an update on the death, and this time he says that there are solid reasons to believe that the event was likely drug related and may have been preventable.
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NK cell biotech Cytovia and gene editing firm Cellectis are expanding upon their previous team-up.
The two biotechs announced yesterday they have expanded their collaboration of gene-edited, iPSC-derived NK and CAR-NK cells to include new CAR targets and development in China by Cytovia’s joint venture entity, CytoLynx Therapeutics.
The amended deal, which was originally agreed to in February, now includes a $20 million equity stake in Cytovia stock by Cellectis, up from $15 million, alongside up to $805 million in milestones and royalty payments.
Zheng Wei, Connect Biopharma CEO
A suite of drugmakers is looking to topple Dupixent in the eczema market — and on Thursday, China-based Connect Biopharma uncorked some mid-stage results it says will pave the way to a pivotal trial. But investors want hard numbers, and without any to share, the company’s stock $CNTB plunged more than 50% on Friday.
Connect’s monoclonal antibody CBP-201 met the primary endpoint in a Phase II trial, showing significant improvements in Eczema Area and Severity Index (EASI) scores in all three dose groups, according to the biotech. The treatment arms — 300 mg every two weeks, 150 mg every two weeks, or 300 mg every four weeks — were all statistically superior to placebo at Week 16, and all showed significant improvements in the proportion of patients achieving at least a 50% or 75% reduction in EASI score (EASI-50 or EASI-75, respectively).
https://endpts.com/fda-opens-up-an-ethical-can-of-worms-with-accelerated-approval-for-first-drug-for-dwarfism/
