US FDA approval tracker: October 2022

 Several drugs got a grilling from the FDA in October, none more so than Covis’s Makena, which is used to reduce preterm birth risk. For a second time, an FDA panel recommended that Makena be removed from the market; the project received accelerated approval in 2011 but its confirmatory study failed in 2019. Now the FDA needs to follow through on its advice, with a final decision expected in the coming months. GSK’s Duvroq, a pill to manage anaemia in chronic kidney disease, had a mixed panel, being recommended for patients on dialysis but not for non-dialysis patients. On the approval front Johnson & Johnson gained a green light for Tecvayli in relapsed/refractory multiple myeloma, although the BCMA bispecific has a Rems program similar to Car-T therapies. Gilead, on the other hand, received a knockback for its hepatitis D project Hepcludex due to concerns over manufacturing and delivery. Gilead gained the project through its $1.4bn buyout of Myr two years ago. Meanwhile, Amicus will have to wait even longer for a decision on its Pompe disease candidate, which was delayed again by the FDA's inability to inspect a plant in China.

Notable first-time US approval decisions in October
Project	Company	Indication(s)	2028e sales by indication ($m)	Outcome
Tecvayli (teclistamab)	Johnson & Johnson	R/r multiple myeloma	1,704	Approved (accelerated)
Hepcludex (bulevirtide)	Gilead/ Hepatera	Chronic hepatitis delta virus infection in adults with compensated liver disease	639	CRL (concerns regarding manufacture & delivery)
Cipaglucosidase for AT-GAA	Amicus	Pompe's disease	266*	Deferred again (alongside second component miglustat; lack of manufacturing site inspection)
SPN-830 apomorphine infusion device	Supernus	Continuous treatment of motor fluctuations (off episodes) in Parkinson’s disease	198	CRL (infusion device issues, inspections)
Tislelizumab	Beigene/ Novartis	2nd-line oesophageal squamous cell carcinoma	83	Pending
Furoscix	Scpharmaceuticals	Congestion in patients with worsening heart failure	-	Approved
Imjudo (tremelimumab) + Imfinzi (Stride regimen)	Astrazeneca	Unresectable hepatocellular carcinoma	-	Approved
*For AT-GAA product. Source: Evaluate Pharma & company releases.

 

Advisory committee meetings in October
Project	Company	Indication	2028e SBI ($m)	Outcome
Duvroq (daprodustat)	GSK	Anaemia due to CKD in adults on/not on dialysis	355	13-3 in favour for patients on dialysis, 5-11 against in non-dialysis patients
Sohonos (palovarotene)	Ipsen	Prevention of heterotopic ossification in adults & children with fibrodysplasia ossificans progressiva	152	Postponed (FDA request for new information on clinical trial data)
Omblastys (omburtamab)	Y-Mabs	Neuroblastoma with CNS/leptomeningeal metastases	69	16-0 against, not provided sufficient evidence to conclude that omburtamab improves OS
Makena	Amag/ Covis	Reducing risk of preterm birth in women who have a history of singleton spontaneous preterm birth	-	14-1 that Makena was not effective, and that it should be removed from the market
Source: Evaluate Pharma, company releases & FDA adcom calendar.

 

Supplementary and other notable approval decisions in October
Product	Company	Indication (clinical trial)	Outcome
Oxlumo	Alnylam	Reduction of plasma oxalate in advanced primary hyperoxaluria type 1 (Illuminate-C)	Approved
Rinvoq	Abbvie	Non-radiographic axial SpA (Select-Axis 2 Study 2)	Approved
Boostrix	GSK	Immunisation during third trimester to prevent pertussis (whooping cough) in infants under 2mths old	Approved
Menveo	GSK	Single-vial presentation to prevent disease caused by meningococcal bacteria serogroups A, C, Y & W in individuals aged 10-55	Approved
mRNA-1273.222 (bivalent)	Moderna	Covid vaccine booster dose, down to six years of age	EUA
Omicron BA.4/BA.5-adapted bivalent Covid-19 vaccine	Pfizer/Biontech	Covid vaccine booster dose, down to five years of age	EUA
Novavax Covid-19 vaccine, adjuvanted (NVX-CoV2373)	Novavax	Covid vaccine booster dose, in adults aged 18 and over (Prevent-19, Cov-Boost)	EUA
Source: Evaluate Pharma & company releases. https://www.evaluate.com/vantage/articles/insights/nme-approvals-snippets/us-fda-approval-tracker-october-2022