A1SLIDES · THE DOSE

The Confirmatory Trial Was Always the Point

Issue 004 · September 2026

In This Issue

In the same week, FDA granted accelerated approval to one oncology regimen and withdrew another indication granted through that pathway. A first treatment for Alexander disease, Lilly's Merida deal and a changed NICE threshold add to the decisions life sciences teams need to explain clearly.

TL;DR

Camizestrant's accelerated approval has a confirmatory obligation still ahead. Krazati's colorectal indication was withdrawn on 1 September. Both show why “FDA approved” is incomplete without the pathway, indication and current evidence status. Beyond oncology, FDA approved the first treatment for Alexander disease, Lilly agreed to buy Merida Biosciences for up to $2.875bn, and NICE's standard cost effectiveness threshold is now £25,000–£35,000 per QALY.

Executive Summary

One pathway produced two different headlines. On 4 September, FDA granted accelerated approval to camizestrant with a CDK4/6 inhibitor for a defined breast cancer population. An April advisory committee had voted 6–3 against its proposed benefit-risk profile. On 1 September, FDA withdrew Krazati with cetuximab's accelerated approval in previously treated KRAS G12C mutated colorectal cancer. Krazati's separate non small cell lung cancer indication remains unaffected.

A rare disease received its first approved treatment. FDA approved Ionis's Zanvastro (zilganersen) for pediatric and adult patients with Alexander disease on 3 September and granted the company a Rare Pediatric Disease Priority Review Voucher.

Capital and access assumptions moved. Lilly agreed to acquire Merida Biosciences for up to $2.875bn, while the UK's standard NICE threshold moved to £25,000–£35,000 per quality adjusted life year from April 2026.

The throughline: an approval or a number is useful only when its conditions, population and current status remain visible.

01 · The Signal

One structural shift. Explained clearly.

Signal

Accelerated approval is a claim with a pending second half: act on earlier evidence, then verify clinical benefit.

The bet extended · Camizestrant

Approval with confirmation still ahead

FDA's Oncologic Drugs Advisory Committee voted 6–3 against the proposed benefit-risk profile for switching to camizestrant when an ESR1 mutation is detected before radiographic progression. FDA granted accelerated approval on 4 September based on the SERENA-6 result. Median progression free survival was 16.0 months versus 9.2 months, measured from detection of the mutation; overall survival data were not mature at that analysis.

The label also carries a boxed warning. Camizestrant taken with ribociclib, or with other QTc-prolonging medicines, can increase the risk of torsades de pointes, other ventricular arrhythmias and sudden death. A slide citing this approval therefore carries two conditions: the pathway obligation and the boxed warning.

Continued approval may depend on verification of clinical benefit in a confirmatory trial. The approval slide therefore needs the pathway and outstanding evidence in the main claim, not only a footnote.

The bet called in · Krazati + Erbitux

A colorectal indication withdrawn

FDA's withdrawn cancer accelerated approvals list records a 1 September 2026 withdrawal for Krazati (adagrasib) with cetuximab in previously treated KRAS G12C mutated colorectal cancer. The combination had received accelerated approval in 2024 on early response data. The confirmatory trial, KRYSTAL-10, compared the combination against chemotherapy in 461 patients with previously treated metastatic disease and did not reach statistical significance on either progression-free or overall survival.

This withdrawal applies to the colorectal combination indication. Krazati's separate non small cell lung cancer indication is unaffected. Teams citing either indication should identify the disease and current regulatory status precisely.

In one independent analysis of 23 selected solid-tumour accelerated approvals, four (roughly 17%) were ultimately withdrawn rather than verified. Withdrawal is a real outcome of the pathway.

Figure 1 · SERENA-6

Median progression free survival

Measured from ESR1 mutation detection.

Figure 2 · ODAC · 30 April 2026

Advisory committee vote

Vote on the proposed benefit risk profile.

Figure 3 · Accelerated approval

One mechanism, two points in its lifecycle

01 / EARLIER EVIDENCE Camizestrant

FDA grants accelerated approval on 4 September. Confirmatory verification remains ahead.

Obligation live
02 / LATER REVIEW Krazati + cetuximab · CRC

FDA withdraws this colorectal indication on 1 September. Its separate NSCLC indication remains.

Indication withdrawn
The same regulatory pathway can lead to different outcomes. Each deck needs the current indication and evidence status.
For life sciences teams

Keep a dated record of every accelerated approval cited in active medical, investor and field decks. Track the indication, label, evidence basis and confirmatory status. Our medical affairs presentation guide covers the update cycle. Teams running internal evidence reviews ahead of an advisory committee meeting may find it useful to structure that session the way an advisory board deck is built.

02 · Capital & Deal Pulse

Notable market movement this September

Eli Lilly × Merida Biosciences · Up to $2.875bn

On 31 August, Lilly agreed to acquire Merida, whose biologics are designed to remove disease causing autoantibodies while preserving normal immune function. Lead candidate MER511 is in Phase 1. The agreement includes an upfront payment and contingent milestones, with closing expected in Q4 2026 subject to customary conditions.

The relevant read through for partner and competitor decks is how Merida's platform broadens Lilly's immunology capabilities. Teams building their own investment story can use our biotech investor deck guide. Read Lilly's announcement.

03 · Pipeline Watch

Regulatory and evidence signals

Approved · Zanvastro for Alexander disease

FDA approved Ionis's Zanvastro (zilganersen) on 3 September for pediatric and adult patients with Alexander disease. It is the first FDA approved treatment for the disease. One population-based estimate puts prevalence at roughly 1 in 2.7 million, and around 500 to 600 cases have been described worldwide. In the pivotal trial, walking speed changed by −2.1% in treated patients versus −35.4% in the control group at week 61, a 33.3 percentage-point difference. Zanvastro targets the abnormal GFAP protein behind the disease. The medicine is administered into the spinal canal every three months. FDA also granted Ionis a Rare Pediatric Disease Priority Review Voucher.

Key signal: the voucher is a distinct strategic asset alongside the clinical approval. Vouchers have changed hands for $150m to $200m in recent transactions, against a reported historical range of $21.2m to $350m since the programme began in 2012. It should be shown as a separate line in launch and value materials.

Regulatory watch · NICE threshold rises

The standard NICE cost effectiveness threshold increased to £25,000–£35,000 per quality adjusted life year from April 2026. Market access teams should review how the updated range applies to their appraisals and economic models, then align field materials with the resulting position. See our HEOR presentation design guide.

On the calendar

Q4 2026 · Expected close of Lilly's Merida acquisition, subject to conditions.

2027 · Expected Europe and Japan submissions for zilganersen through Recordati, according to Ionis.

04 · The Deck

How clinical data actually gets read

Fourth instalment in our running series from The Life Sciences Presentation Report 2026–27. This standard addresses a different kind of omission: presenting a conditional approval as a finished claim.

Standard 04: An accelerated approval has a pending second half. Show both halves, every time.

01

State the exact question a relevant advisory committee voted on and the vote's result.

02

Name the approval pathway explicitly. “FDA approved” alone erases a material distinction.

03

Place the confirmatory trial and its current status beside the approval claim. Use a readout date only when a reliable date is available.

For the visual accuracy principles beneath survival curves and hazard ratios, read our Clinical Data Visualization Playbook.

05 · Editor's View

Perspective from the desk

Put camizestrant and Krazati side by side and the accelerated approval pathway becomes a two part transaction. The agency extends provisional trust on earlier evidence; the sponsor owes confirmation of clinical benefit. Most months, only the approval half is visible in the news cycle. September showed both ends in the same week.

The practical risk is a slide that remains in circulation after the evidence or regulatory status has moved. An approval receives attention immediately. A confirmatory result or indication withdrawal may be less prominent, yet it can change the claim just as decisively.

The fix is a dated review process. Track each accelerated approval cited in active material. Record its indication, evidence basis, confirmatory requirement and current FDA status. When one changes, update the slide and its source line.

About the author

Vaidehi Shukla

Vaidehi Shukla

Principal, Life Sciences & Market Research, Editor for The Dose (UAE) Last updated: September 2026

15 years of life sciences strategy consulting, with leadership roles at IQVIA and Ipsos. Pharmacist by training.

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07 · People Also Ask

Why did FDA approve camizestrant after a negative advisory committee vote?

The advisory committee voted 6–3 against the proposed benefit-risk profile for the early switch. FDA subsequently granted accelerated approval on 4 September 2026, based on progression free survival measured from detection of an ESR1 mutation. Continued approval may depend on verification of clinical benefit in a confirmatory trial.

What happened to Krazati's colorectal cancer indication?

FDA withdrew the accelerated approval for adagrasib with cetuximab in previously treated KRAS G12C mutated colorectal cancer on 1 September 2026. The separate non small cell lung cancer indication remains listed by FDA.

Why is Zanvastro's approval significant?

Zanvastro is the first FDA approved treatment for Alexander disease and the first to target the abnormal GFAP protein that drives it. FDA approved it for pediatric and adult patients on 3 September 2026 and awarded Ionis a Rare Pediatric Disease Priority Review Voucher.

What changed in NICE's cost effectiveness threshold?

The standard threshold rose to £25,000–£35,000 per quality adjusted life year from April 2026. Market access teams should check how the updated range applies to their technology appraisals and economic models.

How should a deck present an accelerated approval?

Name the pathway, the indication, and the evidence on which approval rests. State that clinical benefit still requires confirmation, and keep the confirmatory trial status visible. Recheck active materials when the trial or regulatory status changes.

Sources & Evidence