US approval tracker: December 2022

The FDA granted a flurry of end-of-year approvals, including green lights for Mirati, Gilead and TG Therapeutics. A handful of surprises were also thrown in for good measure, including an early decision for Rigel and Forma’s Rezlidhia in relapsed/refractory IDH1-mutated AML. Grabbing a share of this tiny market will be tough as Servier’s Tibsovo dominates the space. Another surprise was the approval of Adstiladrin, the first gene therapy for bladder cancer, handed to the private group Ferring. Two years ago the project was knocked back by manufacturing issues. Merck & Co’s Keytruda is on the market in the same setting − high-risk BCG-unresponsive non-muscle-invasive bladder cancer. The big pharma also had an early hand in Adstiladrin. In 2011 the project, alongside Merck’s gene therapy portfolio, was handed over to FKD Therapies, with Merck taking an equity stake in the private Finnish company. FKD then signed over Adstiladrin's global commercialisation rights to Ferring in 2018. The approval of Adstiladrin follows other gene therapy thumbs ups in 2022, including those of Skysona and Zynteglo from Bluebird and CSL/Uniqure's Hemgenix.

Notable first-time US approval decisions in December
Project Company Indication(s) 2028e SBI ($m) Outcome
Sunlenca (lenacapavir)  Gilead HIV-1 infection in heavily treatment-experienced people with multidrug-resistant infection  1,602 Approved
Krazati (adagrasib) Mirati/Zai Lab 2L Kras G12C-mutated NSCLC 1,474 Approved (accelerated)
Briumvi (ublituximab) TG Therapeutics Relapsing forms of MS to include clinically isolated
syndrome, relapsing-remitting disease, and active secondary progressive disease, in adults
842 Approved
Tuoyi (toripalimab) Coherus Nasopharyngeal carcinoma 468 Delayed (China facility inspections pending owing to Covid-19 related travel restrictions)
Lunsumio (mosunetuzumab) Roche/Biogen 3L follicular lymphoma 251* Approved (accelerated)
Sohonos (palovarotene) Ipsen Fibrodysplasia ossificans progressiva 152 CRL (additional info on clinical trial data)
Nexobrid Mediwound/Vericel/
Eschar removal (debridement) in adults with deep partial-thickness and/or full-thickness thermal burns 78 Approved
Tislelizumab Beigene/Novartis 2L oesophageal squamous cell carcinoma 83 Pending (Chinese facility inspections)
Olpruva (ACER-001) Acer/Relief Urea cycle disorder 73 Approved
Annik/Anniko (penpulimab) Akeso/Sino 3L nasopharyngeal carcinoma - Pending (Chinese facility inspections)
Rezlidhia (olutasidenib) Rigel/Forma R/r AML with a IDH1 mutation - Approved (2 months early)
Adstiladrin (nadofaragene firadenovec) Ferring (private) Adults with high-risk BCG-unresponsive non-muscle-invasive bladder cancer with carcinoma in situ with or without papillary tumors - Approved
Xenoview (hyperpolarised Xe-129) Polarean Imaging Drug/device combination to evaluate pulmonary function and imaging - Approved
SBI = sales by indication. *SBI not split out. Source: Evaluate Pharma & company releases.


Advisory committee meetings in December
Project Company Indication 2028e SBI ($m) Outcome
Omecamtiv Cytokinetics Reduce the risk of CV death and heart failure events in patients with symptomatic chronic HFrEF 326* 8-3 against
*Forecasts pre-adcom. HFrEF:  heart failure with reduced ejection fraction. Source: Evaluate Pharma, company releases, FDA adcom calendar.
Supplementary and other notable approval decisions in December
Product Company Indication (clinical trial) Outcome
Wegovy Novo Nordisk Chronic weight management in paediatric patients aged 12 years and older with obesity (Step Teens) Approved
Vraylar Abbvie Adjunctive treatment of major depressive disorder (NCT03738215) Approved
Tymlos (abaloparatide) Radius Men with osteoporosis at high risk for fracture (Atom) Approved
Actemra Roche Hospitalised Covid patients (EUA in Jun 2021; Empacta, Covacta, Remdacta, Recovery) Approved
Tecentriq Roche Adult and paediatric patients 2 years of age and older with unresectable or metastatic alveolar soft part sarcoma (Study ML39345) Approved
Idacio (Humira biosimilar) Fresenius Kabi Chronic autoimmune diseases for all eligible indications of the reference product, Humira Approved
Cytalux On Target Laboratories Injectable imaging agent for adults with lung cancer (Elucidate) Approved
Moderna and Pfizer/Biontech bivalent Covid-19 vaccines Moderna, Pfizer/Biontech Covid 19, include use in children down to 6 months of age EUA amended
Abrilada (Humira biosimilar) Pfizer Interchangeability designation Pending
Lynparza + Zytiga + prednisone/prednisolone Astrazeneca Metastatic castration-resistant prostate cancer (Propel) Delayed by 3 months
(Humira biosimilar)
Alvotech/Teva Interchangeability designation CRL (facility reinspection, Pdufa set for April)
Pepaxto Oncopeptides R/r multiple myeloma after at least 4 lines of therapy FDA requested withdrawal of NDA (failed confirmatory Ocean study)
Source: Evaluate Pharma, company releases.

The tables have been updated to include approvals for Olpruva and Wegovy.

For a look at January's upcoming Pdufa decisions, including Eisai's lecanemab see here: Go or no go? Lecanemab’s destiny approaches.

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