RARE stock gained 3.19 percent after FAYUVI approval

Published on 10/05/2026 at 10:56 | Editorial responsibility: Rafael Müller, Editor-in-Chief AD HOC NEWS

RARE stock closed at USD 15.22 on October 2, 2026, after a 3.19 percent daily gain. Q2 revenue reached USD 214 million, while analysts kept a Hold consensus.

RARE, US90400D1081, Illustration mit AI erstellt.

RARE stock (ISIN US90400D1081) closed at USD 15.22 on Nasdaq on October 2, 2026, up 3.19 percent from the prior session. The move followed Ultragenyx Pharmaceutical's September 17 FDA approval of FAYUVI, a gene therapy for Sanfilippo syndrome Type A, as reported by StockTitan.

FAYUVI adds a second gene therapy

According to StockTitan, the FDA granted standard full approval to FAYUVI on September 17, 2026. The treatment is described as the first FDA-approved therapy for the progressive and fatal neurodegenerative disease, and the approval also delivered a Priority Review Voucher.

The regulatory win came after a difficult development update. Ultragenyx reported that its Phase 3 Aspire study of apazunersen in Angelman syndrome missed its primary and key secondary endpoints on September 2, 2026, according to the same StockTitan filing page.

Revenue reaches USD 214 million

Ultragenyx reported second-quarter 2026 revenue of USD 214 million, up 28.5 percent year over year, in the results summarized by MarketBeat. Adjusted loss was USD 0.90 per share against a consensus loss of USD 1.22, while the company reaffirmed 2026 revenue guidance of USD 730 million to USD 760 million.

The quarterly net loss was USD 92 million, compared with USD 115 million in the second quarter of 2025, according to StockTitan. Cash, cash equivalents and marketable securities totaled USD 436 million as of June 30, 2026, while net cash used in operations reached USD 97 million during the quarter.

Analysts see a wide valuation gap

MarketBeat reported a Hold consensus from 21 analysts on September 26, 2026, comprising 10 Hold ratings, 9 Buy ratings, 1 Strong Buy rating and 1 Sell rating. The average 12-month target of USD 34.58 lies 127.2 percent above the October 2 price of USD 15.22.

Recent analyst actions remained mixed. Cantor Fitzgerald raised its target from USD 33 to USD 39 and kept an Overweight rating on September 18, 2026, while Citigroup lifted its target from USD 31 to USD 32 and retained Buy, according to Yahoo Finance.

RARE remains below its yearly high

At USD 15.22 on October 2, 2026, RARE stood 61.8 percent below its 52-week high of USD 39.89 and above its 52-week low of USD 12.73. The next valuation test is whether FAYUVI commercialization can convert regulatory progress into revenue while the company works toward profitability in 2027.