Go or no go? Pdufa resolutions eyed for J&J and Amryt

February will also see an outcome for Gilead's long-acting HIV therapy, and a key FDA panel for me-too checkpoint MAbs.

Several projects with delayed Pdufa decisions should finally see outcomes next month, including J&J/Legend's ciltacabtagene autoleucel and Amryt’s Oleogel-S10. Immunocore, however, was able to grasp an early approval today for its immunotherapy tebentafusp, now called Kimmtrak, nearly a month before its February Pdufa decision.

One of the most important regulatory discussions in February will come from a panel meeting on Lilly and Innovent’s Tyvyt. The adcom could indicate whether data generated in China can back US approval. 

Tyvyt’s China study, Orient-11, tested the anti-PD-1 MAb in combination with chemotherapy in first-line non-squamous NSCLC. While the results showed a survival benefit the comparator was chemo alone and not Keytruda  the Merck & Co drug that is redefining front-line NSCLC therapy, based on the Keynote-189 study. 

If the regulators decide that head-to-head US studies are needed here Tyvyt, and others relying on China data, could be in for long delays (Days of reckoning for immune checkpoint blockers, January 4, 2022). 

Three months later... 

J&J/Legend’s BCMA-targeted Car-T project cilta-cel should finally get a decision next month; its original Pdufa date was November, but this was pushed back to give the FDA time to review new information on "an updated analytical method following an FDA information request", according to J&J.

Cilta-cel has demonstrated a strong clinical profile in heavily pretreated multiple myeloma patients, and boasts better efficacy on a cross-trial basis than Bristol Myers Squibb/Bluebird's Abecma, which was approved for fifth-line use last year. 

Cilta-cel's label will likely echo Abecma’s, which includes a black box warning of haemophagocytic lymphohistiocytosis/macrophage activation syndrome and cytopenia, in addition to the cytokine release syndrome and neurotoxicities that are standard with Car-T therapies.  

Also suffering a delay was Amryt’s epidermolysis bullosa project Oleogel-S10, with the regulators needing extra time to review additional data analyses. 

Despite the delay Oleogel-S10, a topical gel formulation of the plant extract betulin, could still become the first therapy for the rare skin disorder, but Krystal Biotech is gaining ground. Krystal’s topical gene therapy Vyjuvek is expected to be filed in the first half of the year; the asset recently yielded promising topline data, although there are still unanswered questions.

Long acting 

Meanwhile, Gilead’s lenacapavir could become the first capsid inhibitor approved for HIV, and the only treatment given every six months. A green light is likely by the end of next month in heavily pretreated patients, but a push into earlier treatment lines will be key to this project's chances of generating big bucks. 

The phase 2/3 Capella study found that 88% of lenacapavir-treated patients achieved a clinically meaningful viral load reduction of at least 0.5log10 copies/ml, versus 17% of patients receiving placebo. Here the focus was heavily treatment-experienced patients with multidrug-resistant HIV who were failing on antiretroviral drugs (Gilead challenges Viiv in heavily treated HIV, March 10, 2021). 

Viiv’s Rukobia is already approved in heavily treatment-experienced patients, but is given as a twice-daily tablet.

Phase 3 trials with lenacapavir in pre-exposure prophylaxis have already started. However, these and other studies were put on clinical hold in December owing to vial contamination concerns

And Viiv again is one step ahead with Apretude – the extended-release version of cabotegravir got the green light in December for Prep, where it is given every two months after an initial loading dose.

The tables below list first-time and supplementary US approvals, as well as advisory committee meetings due next month, with consensus forecasts from Evaluate Pharma

Notable first-time US approval decisions due in February
Project Company Pdufa date Indication(s) 2026e sales by indication ($m) Note
(immune globulin intravenous, 10% liquid)
GC Pharma Feb Primary humoral immunodeficiency - -
Priorix Glaxosmithkline Est Feb MMR vaccine 364 Gained EMA approval back in 1997, filed in the US in Aug 2021
Sutimlimab Sanofi Feb 2 (resubmitted) Haemolysis in adults with cold agglutinin disease 438 Previous CRL due to deficiencies at manufacturing facility
Mitapivat Agios Feb 17 Pyruvate kinase deficiency 287 Also in development for thalassaemia and sickle cell disease
Bardoxolone Reata Feb 25 Alport syndrome 1,210* Negative adcom in December
d-amphetamine transdermal system
Est Feb 25 ADHD - -
Amryt Feb 28 Junctional and dystrophic epidermolysis bullosa 269 Extended from Nov by submission of additional analyses
Ciltacabtagene autoleucel
Johnson & Johnson/
Legend Biotech
Feb 28 3L+ multiple myeloma 1,692 Extended from Nov by info request on analytical method
CTI Biopharma Feb 28 Myelofibrosis patients with severe thrombocytopenia 368 Extended from Nov after new of data submission
Lenacapavir Gilead Feb 28 Multidrug-resistant HIV in heavily treatment-experienced patients  877 See text
Akeso/Sino Est H1 3L nasopharyngeal carcinoma - Days of reckoning for immune checkpoint blockers
177Lu-PSMA-617  Novartis H1 Radioligand therapy for mCRPC 851 Asco 2021 justifies Novartis’s radioligand Vision
*Forecasts made before Dec adcom. Source: Evaluate Pharma & company releases.


Advisory committee meetings in February
Project Company Adcom date Indication Note
Tyvyt (sintilimab) +
pemetrexed +
platinum chemo 
Feb 10 1L stage IIIB, IIIC, or Stage IV non-squamous NSCLC Days of reckoning for immune checkpoint blockers, Pdufa in March
IV tramadol Avenue Feb 15 Acute pain Tentatively scheduled, notified in PR but no official notice on FDA website, two previous CRLs
Comiranty Pfizer/Biontech Feb 15 Amend the EUA for administration to children 6 months through 4 years of age Already authorised for use in children aged 5-11 years, also as a single booster dose in individuals 12-15 years and older
Source: Evaluate Pharma & FDA adcom calendar.


Supplementary and other notable approval decisions in February
Product Company Indication (clinical trial) Date
Kymriah Novartis r/r FL after two prior lines of treatment (Ph2 Elara) Est Feb 25
Lynparza Astrazeneca Adjuvant BRCA-mutated Her2-negative breast cancer (OlympiA) Q1
Fasenra Astrazeneca Nasal polyps H1
Olumiant Lilly Atopic dermatitis (Breeze-AD programme) Pending
Rinvoq Abbvie Ankylosing spondylitis and ulcerative colitis  Pending
Source: Evaluate Pharma & company releases. 

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