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• The FDA granted approval to Dupixent (dupilumab) for treating patients aged 6 years and up with allergic fungal rhinosinusitis and a history of sino-nasal surgery, marking the first treatment approved for this indication, according to a press release.

• The release noted that the FDA’s decision on Dupixent was, in part, based on findings from the LIBERTY-AFRS-AIMS trial.

“Before Dupixent, people living with allergic fungal rhinosinusitis had to rely on treatments that left them potentially vulnerable to regrowth of nasal polyps and thick mucus that could rob them of their sense of smell,” Alyssa Johnsen, MD, PhD, global therapeutic area head, immunology development at Sanofi, said in the release. (Healio)

• Swedish biotech Immedica won an accelerated approval from the FDA, resurrecting the first treatment to address persistently elevated levels of plasma arginine, which is the primary driver of the rare disease ARG1-D.

• The approval of Loargys, an arginine-specific enzyme, follows a complete response letter in August and Immedica’s acquisition of the compound in 2022 after the bankruptcy of Aeglea BioTherapeutics.

• Aeglea in 2022 received a refuse-to-file letter from the FDA, which sought more “evidence showing that plasma arginine and metabolite reduction predicts clinical benefit in patients with ARG1-D.”

• Immedica will win a priority review voucher as part of the approval, which could provide the KKR-backed biotech with $100 million to $200 million when it’s sold. (Endpoints)

• AstraZeneca secured a key regulatory win in its effort to reclaim the lead in the BTK inhibitor market, with the FDA approving its combination of Calquence plus Venclexta as the first all-oral, fixed-duration regimen for first-line chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL).

• While patients with newly diagnosed CLL typically take a BTK drug like Calquence indefinitely until disease progression, the new approval offers a better quality-of-life alternative by limiting treatment to 14 months.

“The continuous regimens frequently used to treat chronic lymphocytic leukemia often come with side effects that may become burdensome to patients over time,” Jennifer Brown, MD, PhD, from the Dana-Farber Cancer Institute, said in a statement, adding that the Calquence combo gives doctors greater flexibility to tailor treatment plans. (Fierce Pharma)

• After concluding last year on solid footing with FDA approval of its motion sickness drug Nereus (tradipitant), Vanda announced that the regulator has now authorized the atypical oral antipsychotic Bysanti (milsaperidone) for the acute treatment of manic or mixed episodes associated with bipolar I disorder and for schizophrenia in adults.

• Vanda explained that Bysanti builds on its well-established therapy Fanapt (iloperidone), approved earlier for the same indications.

• The therapy, which is likely to launch in the third quarter, quickly interconverts to Fanapt in vivo, producing dual active molecules that antagonise dopamine D2, serotonin 5-HT2A and alpha1-adrenergic receptors.

• The company noted that in clinical studies Bysanti demonstrated bioequivalence to Fanapt, with its NDA filing last year supported by the latter’s existing clinical data, including two acute schizophrenia studies, one bipolar I study, and a schizophrenia relapse-prevention trial. (FirstWord Pharma)

• Treatments with a similar mechanism could be grouped together to allow for a single approval that can be adapted based on different genetic mutations, for example.

• Only a month after Jazz Pharmaceuticals said it had signed a deal to sell an FDA priority review voucher (PRV) for $200 million, a new PRV transaction involving Fortress Biotech and an unnamed buyer shows that the trend of rising voucher prices is still going strong.

• On February 23, Fortress said its subsidiary, Cyprium Therapeutics, has entered into an agreement to sell a recently received rare pediatric disease priority review voucher for $205 million.

• Cyprium got the PRV as part of the FDA’s recent approval of injected copper replacement therapy Zycubo as the first treatment approved in the US for the rare neurodegenerative disorder Menkes disease.

• While another company, Sentynl Therapeutics, is handling development and commercialization of Zycubo under a 2023 agreement, the deal called for Sentynl to transfer the PRV back to Fortress/Cyprium after the approval. (Fierce Pharma)

• Building up its base of medicines for lung diseases, GSK said Wednesday it was buying the privately held firm 35Pharma for $950 million in cash.

• The centerpiece of the deal is an experimental drug called HS235 that is set to start trials in pulmonary arterial hypertension, a form of high blood pressure in the lungs.

• The disease has increasingly become a target for drugmakers in recent years. Merck’s Winrevair won approval for PAH in 2024, and reached $1.4 billion in sales last year.

• 35Pharma, based in Canada, has completed Phase 1 safety trials of HS235. In addition to the PAH trial, GSK plans to test it in a related disease called pulmonary hypertension due to heart failure with preserved ejection fraction. (STAT)

• Roche has started looking for a partner to handle production and supply of Rocephin after concluding it is becoming “increasingly unsustainable” to make the antibiotic in Switzerland.

• Rocephin is Roche’s brand name for ceftriaxone, a long-acting, broad spectrum cephalosporin antibiotic. Since ceftriaxone was approved in the 1980s, physicians have used the injectable antibiotic to treat conditions such as sepsis and meningitis in children and adults. The antibiotic is on the World Health Organization’s list of essential medicines.

• Roche serves the market from its production site in Kaiseraugst, Switzerland. Yet after manufacturing the antibiotic for 40 years, Roche is now seeking a production partner. (BioSpace)

“These therapies are incredible,” says Dr. Will Shrank, a former Harvard Medical School professor who’s also worked for insurance companies such as Humana and CVS Health.

“They can absolutely massively impact the lives of patients with terrible conditions that, when I was in medical school, none of us could imagine were curable or addressable.”

“The fragmented way that we pay for and deliver care in this country is perfectly misaligned with equitably delivering those therapies to patients who can benefit,” says Shrank.

• Shrank recently founded a company called Aradigm to help make gene therapy more widely available.

• State Medicaid programs in Nebraska, North and South Dakota and Oklahoma paid for relatively few gene therapies, for example.

• Meanwhile, states including California, Indiana, Massachusetts and Texas paid for more.

• In theory, even if someone’s state doesn’t have a hospital that offers gene therapy, the state Medicaid program is supposed to pay for that person to get it in another state, Sarpatwari says.

• But NPR’s analysis shows that isn’t happening, he says, “and that’s troubling.” (NPR)