US FDA approval tracker: August 2022

It was a double FDA thumbs-up for Astrazeneca and Daiichi Sankyo’s Enhertu last month as the antibody-drug conjugate became the first Her2-targeted agent approved in Her2-positive lung cancer and Her2-low breast cancer. Enhertu looks set to dominate the Her2-low space, a newly defined subset in breast cancer, as competitors are still some way behind. Bluebird also received approval for Zynteglo, a gene therapy for transfusion-dependent beta-thalassaemia, but the company has a huge commercial task ahead to try and make the product profitable. Bluebird’s second gene therapy, eli-cel, has a Pdufa date in September, this time for cerebral adrenoleukodystrophy, a rare metabolic disorder. Investors will have to wait a little longer for several pending FDA decisions, including J&J’s multiple myeloma bispecific Tecvayli, which gained European approval in fourth-line disease last month. 

Notable first-time US approval decisions in August
Project Company Indication(s) 2028e sales by indication (SBI) ($m) Outcome
Axsome Major depressive disorder 787 Approved
Cormedix Prevention of catheter-related bloodstream infections  392 CRL (second, this time due to deficiencies at CMO)
Xenpozyme Sanofi Non-CNS manifestations of acid sphingomyelinase deficiency (Niemann-Pick disease) in adult and paediatric patients 346 Approved (~1 month early)
Miglustat for AT-GAA Amicus Pompe disease 266* No decision yet (part of two-component therapy with cipaglucosidase, Pdufa in Oct)
Tecvayli (teclistamab) Johnson & Johnson R/R multiple myeloma  260 No decision yet
Betibeglogene autotemcel (Zynteglo/ beti-cel) Bluebird Beta-thalassaemia (pts requiring regular red blood cell transfusions) 120 Approved
Libervant (diazepam) buccal film Aquestive Acute treatment of intermittent, stereotypic episodes of frequent seizure activity 90 Tentative approval (Neurelis' Valtoco has orphan drug exclusivity)
Annik (penpulimab) Akeso/Sino 3L nasopharyngeal carcinoma - No decision yet
*Forecast for AT-GAA. Source: company releases & Evaluate Pharma.
Private companies with pending FDA approvals
Project Company Indication Outcome
SH-111 Shorla Oncology Undisclosed project for T-cell leukaemia Pending (was filed in Apr 2021)
Spesolimab Boehringer Ingelheim Generalised pustular psoriasis Decision expected by YE
Source: company releases & Evaluate Pharma.


Supplementary and other notable approval decisions in August
Product Company Indication (clinical trial) Outcome
Nuplazid Acadia Hallucinations and delusions associated with Alzheimer's disease psychosis (Harmony, phase 2) CRL (new study required)
Myfembree Pfizer/
Moderate-to-severe pain associated with endometriosis (Spirit 1, Spirit 2) Approved
Enhertu Astra/Daiichi Sankyo 2L Her2-positive NSCLC (Destiny-Lung01, Destiny-Lung02) Approved (accelerated)
Unresectable metastatic Her2 low breast cancer (Destiny-Breast04) Approved (~2 months early)
Cimerli (CHS-201/ FYB201, Lucentis biosimilar) Teva/Bioeq/
Wet AMD, macular oedema following retinal vein occlusion, DME, diabetic retinopathy, and myopic choroidal neovascularisation Approved
Haloette (generic Nuvaring) Mayne Pharma/
Vaginal hormonal contraceptive ring Approved (three previous CRLs)
Nubeqa + docetaxel Bayer/
Metastatic hormone-sensitive prostate cancer (Arasens) Approved
Calquence tablet Astrazeneca Adult patients with chronic lymphocytic leukaemia, small lymphocytic lymphoma and for patients with relapsed or refractory mantle cell lymphoma (Elevate-Plus) Approved
Xofluza Roche Treatment of influenza in children aged 5-12, and for the prevention of influenza following contact with someone with influenza (oral therapy) (Ministone-2, Blockstone) Approved
Hadlima (High concentration Humira biosimilar) (SB5) Samsung Bioepis/Organon RA, juvenile idiopathic arthritis, psoriatic arthritis, ankylosing spondylitis, Crohn's, ulcerative colitis, plaque psoriasis (NCT04514796) Approved (launch expected on/after July 1 2023)
Tabrecta Novartis Adult patients with metastatic NSCLC whose tumours have a mutation leading to MET exon 14 skipping (Geometry mono-1) Full approval (accelerated approval granted 2020)
Pemazyre Incyte Myeloid/lymphoid neoplasms with FGFR1 rearrangement (Ph2 Fight-203) Approved
Imbruvica J&J Paediatric patients one year and older with chronic graft-versus-host disease after failure of one or more lines of systemic therapy (Ph1/2 Imagine) Approved
Source: company releases & Evaluate Pharma.


Product Company Indication/outcome
Jynneos Bavarian Nordic Approved for use in individuals determined to be at high risk for monkeypox infection
Nuvaxovid Novavax Prevention in Covid-19 in individuals aged 12-17 years old (vaccine, first granted an EUA in July)
Bivalent Covid-19 vaccine (mRNA-1273.222) Moderna For use as a booster dose, includes original Sars-Cov-2 strain and omicron variant
Bivalent Covid-19 vaccine Pfizer/Biontech
Source: FDA, company releases & Evaluate Pharma.

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