Shots:
Ultragenyx has entered into a definitive agreement to sell a Rare Pediatric Disease PRV for $210M, provide Ultragenyx with significant non-dilutive capital to advance therapies for rare & ultra-rare diseases
Rare Pediatric Disease PRV was received following FDA approval of Genglycos (pariglasgene brecaparvovec-opnr), the first treatment targeting the underlying cause of glycogen storage disease…
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Italfarmaco & VeriSIM Life have entered into a strategic collaboration to discover & develop novel histone deacetylase (HDAC)-targeted therapeutic assets for neuromuscular, fibrotic, & rare diseases
As per the deal, VeriSIM Life will receive technology service fees, development, regulatory, & commercial milestone payments, and royalties on future commercial products, with the total potential value…
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Shionogi announced a planned acquisition of IntraBio for $2.0B, adding AQNEURSA (levacetylleucine) and IntraBio’s rare disease capabilities to its portfolio; the transaction is expected to close between November and December 2026, subject to customary conditions and regulatory approvals
AQNEURSA is approved in the US and EU for neurological manifestations of Niemann-Pick disease Type C…
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Belite Bio submitted a New Drug Application (NDA) to Japan’s MHLW for once-daily oral tinlarebant (LBS-008) for the treatment of Stargardt disease type 1 (STGD1)
The NDA was submitted under Japan’s Sakigake Designation System, following tinlarebant’s designation as a Sakigake pharmaceutical product; if approved, it could become the first approved treatment for STGD1 and…
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Ultragenyx reported the US FDA accelerated approval to GENGLYCOS (pariglasgene brecaparvovec-opnr; DTX401) for adults and pediatric patients (≥8yrs., n=46) with glycogen storage disease type Ia (GSDIa)
The approval was supported by the P-III (GlucoGene) study, in which GENGLYCOS (n=20) demonstrated a statistically significant reduction in cornstarch requirements at 48wks. vs PBO (n=24) (p<0.001). The…
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Alfasigma has entered into an agreement with SEVER Life Sciences to acquire 100% of Nordic Pharma & its subsidiaries, adding the Nordimet methotrexate franchise to Alfasigma’s rheumatology portfolio alongside Jyseleca
Deal will expand Alfasigma’s commercial footprint through Nordic Pharma’s direct operations across 18 EU countries, while adding platforms in Canada & Japan. The portfolio…
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The EC has granted ODD to ABO-101 for the treatment of primary hyperoxaluria
ABO-101 is being evaluated in an open-label global P-I/II (redePHine) study assessing safety, tolerability, PK/PD, & preliminary efficacy of a single dose of ABO-101 in pts with PH type 1
ABO-101 is a one-time, liver-directed gene-editing therapy that uses an Acuitas-licensed…
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Could a targeted biologic therapy help address the longstanding unmet needs in hypereosinophilic syndrome (HES)? AstraZeneca’s FASENRA has now received US approval for HES, introducing a once-monthly treatment option for patients living with this rare and often underdiagnosed eosinophilic disorder
Results from the Phase III NATRON trial showed that FASENRA reduced the risk of first HES flare by 65% vs placebo,…
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A consortium led by CVC and GBL launched a voluntary cash tender offer to acquire Recordati at €51.29/share, valuing the transaction at ~€10.7B (~$12.4B). The offer aims to delist Recordati from Euronext Milan & is expected to close in Q4’26
The offer is backed by Rossini, which agreed to tender its 97.9M shares (~46.8%…
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Angelini Pharma has entered into a definitive agreement to acquire all outstanding shares of Catalyst, marking Angelini’s entry into the US market
As per the deal, Angelini will acquire Catalyst for $31.5 per share in cash, for a total equity value of ~$4.1B; closing is expected in Q3’26
The acquisition will strengthen Angelini’s Brain Health…

