Regulatory channel feed

Hal Barron at Endpoints News' UKBIO 2019

FDA ap­proves GSK's BC­MA drug, mark­ing a rocky re­turn to can­cer for the British gi­ant

Despite concerns on both safety and efficacy, the FDA approved GlaxoSmithKline’s multiple myeloma drug belantamab mafodotin, deciding that for the sickest patients the benefits outweighed the risk. It will be marketed as Blenrep and sold for $23,900 per month.

The approval is notable both for the multiple myeloma field and for GSK itself. It’s the first approval for a drug that targets BCMA, a protein overexpressed on multiple myeloma cells that researchers have been trying to target for over a decade. A flurry of other BCMA drugs, having already shown promise in early trials, are likely to follow in the next few years.

Stephen Hahn, FDA commissioner (AP Images)

FDA touts ad­vanced man­u­fac­tur­ing to ad­dress Covid-19 short­falls

Advanced manufacturing techniques can be employed to help address some of the manufacturing and supply chain problems the US has seen during the Covid-19 pandemic, Stephen Hahn, FDA commissioner, and Anand Shah, FDA deputy commissioner for medical and scientific affairs, wrote in a blog post on the FDA website.

“The potential public health value of advanced manufacturing is even greater in the context of the ongoing COVID-19 pandemic, which has highlighted the strain on supply chains and the need for adaptive manufacturing systems to accelerate the production of medical countermeasures,” Hahn and Shah wrote. “The FDA has established a strong regulatory foundation to support the uptake of advanced manufacturing, and COVID-19 provides the unique impetus to spur further advancement of medical manufacturing.”

Long jour­ney for DBV peanut patch ends in re­jec­tion by FDA

DBV Technologies’ long and winding saga with the FDA has ended — for now, at least — with a CRL for their peanut allergy skin patch.

The rejection comes 21 months after the company first filed for approval. At the time, the French biotech was in a two-way race with California’s Aimmune to produce the first FDA-approved treatment for peanut allergy. But since then Aimmune cleared that hurdle, while DBV, after a withdrawal and resubmission, indicated that communication between them and regulators had broken down.  In June, the company began cutting programs and employees.

J&J gets a fresh OK for es­ke­t­a­mine, but is it re­al­ly the game-chang­er for de­pres­sion Trump keeps tweet­ing about?

Backed by an enthusiastic set of tweets from President Trump and a landmark OK for depression, J&J scooped up a new approval from the FDA for Spravato today. But this latest advance will likely bring fresh scrutiny to a drug that’s spurred some serious questions about the data, as well as the price.

First, the approval.

Regulators stamped their OK on the use of Spravato — developed as esketamine, a nasal spray version of the party drug Special K or ketamine — for patients suffering from major depressive disorder with acute suicidal ideation or behavior.

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FDA hands Mor­phoSys and In­cyte a quick OK on their po­ten­tial block­buster CAR-T al­ter­na­tive

Nearly three years after okaying the CAR-Ts Yescarta and Kymriah, the FDA has approved a new CD19 therapy.

MorphoSys’ Monjuvi, or tafasitamab-cxix, was cleared Friday for use in refractory diffuse large B-cell lymphoma (DBLCL). The approval sets up both MorphoSys and their commercial partner Incyte to compete with Gilead and Novartis in the ultra-competitive indication, where similar trial results and far easier delivery could allow them to cut a fair share of the market.

Levi Garraway, Roche CMO

Roche snags an FDA ap­proval for an ad­vanced melanoma drug cock­tail — but it's up against some fierce com­pe­ti­tion

Roche’s PD-L1 inhibitor Tecentriq snagged an FDA approval to treat certain advanced melanoma patients in a three-drug cocktail, providing another option for patients with a typically poor prognosis.

In a Phase III IMspire150 study, Tecentriq combined with Cotellic (cobimetinib) and Zelboraf (vemurafenib) significantly beat a placebo mix in extending progression-free survival in BRAF V600 mutation-positive advanced melanoma patients. Participants on Tecentriq lived a median 15.1 months without worsening disease, compared to 10.6 months in patients on Cotellic, Zelboraf, and the placebo.

Gly­col­ic acid, TCA among FDA's pro­posed ad­di­tions to 503B bulk drugs list

Four new bulk substances are up for inclusion on the list of active pharmaceutical ingredients that outsourcing facilities can use in drug compounding under section 503B of the Federal Food, Drug, and Cosmetic Act, according to a proposal from the FDA.

The four substances FDA is proposing to include as bulk drug substances are diphenylcyclopropenone, glycolic acid, squaric acid dibutyl ester and trichloroacetic acid. An additional 19 bulk drug substances were considered for inclusion in the 503B bulk drug substances list, but FDA is proposing not to include these substances, citing a lack of clinical need for an outsourcing facility to compound drugs from the 19 substances. This lack of need, said the agency, meant the drugs did not meet the statutory criteria for addition to the list.

House ap­proves $3.2 bil­lion FDA bud­get

The House of Representatives passed a four-bill appropriations minibus for fiscal year 2021 that includes an amendment giving the FDA the authority to recall drugs. The agency’s proposed budget also includes targeted boosts to spending for medical product and food safety activities and influenza vaccine manufacturing technologies.

Other targeted initiatives include cross-departmental initiatives designed to enhance food and medical product safety; additionally, “[T]he bill includes a strong focus on continuing FDA’s efforts to enable faster responses to foodborne illness outbreaks and increase the safety and cybersecurity of medical devices,” according to the House Appropriations Committee’s summary of the minibus.

Ilan Ganot, Solid Bio CEO (Solid Bio)

FDA keeps Sol­id Bio's trou­bled Duchenne MD gene ther­a­py on clin­i­cal lock­down in lat­est set­back

Much-maligned Solid Biosciences received yet another spate of bad news late Friday regarding its Duchenne muscular dystrophy gene therapy program.

It’s been almost nine months since the FDA announced a second clinical hold on Solid Bio’s Phase I/II trial for an AAV-based Duchenne MD treatment. And after extending that hold in May, US regulators again decided they needed more info with another extension on Friday.

Ken Takeshita, Kite global head of clinical development (Gilead)

Gilead’s Kite snares a land­mark FDA OK on a $373,000 CAR-T ther­a­py — which comes with brag­ging rights to the first true fran­chise op

While many of the top players in the immuno-oncology field have shifted their focus to new and better CAR-Ts in the pipeline, one of the pioneers in the field is now proudly laying claim to a landmark achievement — gaining a second OK and creating the first commercial CAR-T portfolio.

“We’re about to become the first company with a cell therapy franchise,” says Ken Takeshita, head of global development at Kite Therapeutics, a subsidiary of Gilead. The drug will be marketed as Tecartus and sold for $373,000, according to a company spokesperson on Friday.

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