RAREHIGH SIGNALREGULATORY10-K

RARE received an Incomplete Response Letter from the FDA in February 2026 for its resubmitted BLA for UX111, requesting additional CMC documentation after the company had submitted substantial longer-term neurologic data.

The IRL represents a significant setback for RARE's lead gene therapy candidate, extending the approval timeline further after the initial Complete Response Letter in July 2025. While the FDA's request appears focused on chemistry, manufacturing, and controls documentation rather than clinical efficacy concerns, this delay prolongs uncertainty around a key pipeline asset that could drive future growth for the rare disease specialist.

Comparing 2026-02-18 vs 2025-02-19View on EDGAR →
FINANCIAL ANALYSIS

RARE demonstrated solid operational momentum with revenue growing 20% to $673M, supported by expanding receivables and inventory levels that suggest strong commercial demand for its rare disease portfolio. However, the company's cash burn intensified with operating cash flow declining to -$466M from -$414M, while total liabilities increased meaningfully to $1.6B. The financial profile reflects a company investing heavily in pipeline development while generating growing commercial revenues, though the regulatory setback may pressure near-term investment priorities.

FINANCIAL STATEMENT CHANGES
Accounts Receivable
Balance Sheet
+29.7%
$121.8M→$158.0M

Receivables grew 29.7% — monitor days sales outstanding for collection efficiency.

Total Liabilities
Balance Sheet
+29.3%
$1.2B→$1.6B

Liabilities increased 29.3% — monitor debt-to-equity ratio and interest coverage.

Revenue
P&L
+20.1%
$560.2M→$673.0M

Revenue growing 20.1% — solid top-line momentum, watch margins for quality of growth.

Current Assets
Balance Sheet
+16.4%
$817.1M→$951.0M

Current assets grew 16.4% — improving short-term liquidity or inventory/receivables build.

Inventory
Balance Sheet
+15.5%
$45.0M→$52.0M

Inventory built 15.5% — monitor whether demand supports this build or if write-downs may follow.

Operating Cash Flow
Cash Flow
-12.5%
-$414.2M→-$466.0M

Operating cash flow softened — monitor whether temporary working capital timing or structural deterioration.

Current Liabilities
Balance Sheet
+11.6%
$344.2M→$384.0M

Current liabilities rose 11.6% — increased short-term obligations, watch current ratio.

LANGUAGE CHANGES
NEW — 2026-02-18
PRIOR — 2025-02-19
ADDED
As of February 13, 2026, the Company had 96,629,788 shares of common stock issued and outstanding.
In January 2026, we resubmitted our BLA seeking accelerated approval for UX111 (rebisufligene etisparvovec) as a treatment for patients with Sanfilippo syndrome Type A (MPS IIIA) to the FDA.
The submission included substantial longer-term data on multiple measures of neurologic benefit to support an intermediate clinical endpoint for accelerated approval supported further by CSF heparan sulfate and other biomarker data, as agreed with the FDA during the last clinical review.
In February 2026, we received an Incomplete Response Letter, or IRL, from the FDA regarding our resubmitted BLA.
The IRL requested additional supportive documentation related to our CMC responses to the CRL we received in July 2025.
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REMOVED
As of February 13, 2025, the Company had 92,501,126 shares of common stock issued and outstanding.
We have four commercially approved products, Crysvita (burosumab) for the treatment of X-linked hypophosphatemia, or XLH, and tumor-induced osteomalacia, or TIO, Mepsevii (vestronidase alfa) for the treatment of mucopolysaccharidosis VII, or MPSVII or Sly Syndrome, Dojolvi (triheptanoin) for the treatment of long-chain fatty acid oxidation disorders, or LC-FAOD, and Evkeeza (evinacumab) for the treatment of homozygous familial hypercholesterolemia, or HoFH.
In November 2024, we announced that we had received a positive finalized assessment report with agreement to file for Conditional Early Approval, or CEA, from Japan s Pharmaceuticals and Medical Devices Agency, or PMDA, based on the currently available global clinical data for the product.
With this feedback, we expect to file a Japan-New Drug Application for CEA in mid-2025.
Food and Drug Administration, or FDA, and European Medicines Agency, or EMA, Rare Pediatric Disease designation and Breakthrough Designation from the FDA, and was accepted into the EMA s Priority Medicines, or PRIME, program.
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